The US FDA approved 53 new drug therapies in 2020. The new drug therapies Active ingredient, approval date, intended use, drug use, and the benefits from the FDA portal is given below –
Drug Name: Gemtesa
Active Ingredient: vibegron
Approval Date: 12/23/2020
FDA-approved use: To treat overactive bladder
Drug Use: GEMTESA is used treat the following symptoms due to a condition called overactive bladder (OAB): a strong need to urinate with leaking or wetting accidents, the need to urinate right away, and the need to urinate often. GEMTESA is a tablet taken my mouth once a day. After 12 weeks of treatment, patients who received GEMTESA experienced reduced number of OAB related symptoms in comparison to patients who received placebo.
Drug Name: Ebanga
Active Ingredient: ansuvimab-zykl
Approval Date: 12/21/2020
FDA-approved use: To treat ebola
Drug Name: Orgovyx
Active Ingredient: relugolix
Approval Date: 12/18/2020
FDA-approved use: To treat advanced prostate cancer
Drug Use: ORGOVYX is a drug for the treatment of adults with advanced prostate cancer. ORGOVYX is a tablet. Three tablets (total of 360 mg) are taken on the first day of treatment followed by one tablet once daily. Because prostate cancer cells use testosterone for growth, the trial tested the ability of ORGOVYX to keep testosterone level as low as after castration. In this trial, 97% of all patients treated with ORGOVYX reached and maintained low testosterone levels equivalent to castration level.
Drug Name: Margenza
Active Ingredient: margetuximab (anti-HER2 mAb
Approval Date: 12/16/2020
FDA-approved use: To treat HER2+ breast cancer
Drug Use: MARGENZA is a drug for treatment of metastatic HER2-positive breast cancer in adult patients who have been previously treated for their metastatic disease. MARGENZA is an injection. It is given by a healthcare provider directly into the vein (intravenous infusion) every three weeks in combination with chemotherapy. Patients who received MARGENZA in combination with chemotherapy lived longer without cancer progression in comparison to patients who received an approved drug trastuzumab in combination with chemotherapy.
Drug Name: Klisyri
Active Ingredient: tirbanibulin
Approval Date: 12/14/2020
FDA-approved use: To treat actinic Keratosis of the face or scalp
Drug Use: KLISYRI is used on the skin to treat patients with actinic keratosis on the face or scalp. KLISYRI is an ointment. One package of ointment is applied on the affected area of face or scalp 1 time a day for 5 days in a row. More patients achieved complete clearance of AK after 5 days of treatment with KLISYRI in comparison to those who were treated with control ointment (vehicle).
Drug Name: Orladeyo
Active Ingredient: berotralstat
Approval Date: 12/03/2020
FDA-approved use: To treat patients with hereditary angioedema
Drug Use: ORLADEYO is a drug used to prevent attacks of hereditary angioedema (HAE) in patients 12 years and older. ORLADEYO is a capsule taken by mouth, 1 time every day with food. Patients who received ORLADEYO had fewer angioedema attacks compared to those receiving the placebo.
Drug Name: Gallium 68 PSMA-11
Active Ingredient: Gallium 68 PSMA-11
Approval Date: 12/01/2020
FDA-approved use: For detection and localization of prostate cancer
Drug Use: Ga 68 PSMA-11 is a drug used for detection of specific cancer lesions in men with prostate cancer – whose newly diagnosed cancer could be cured with the initial treatment, or, who have been treated for prostate cancer but have high prostate-specific antigen (PSA) in their blood. High PSA in the blood of these patients is a suspicious sign that cancer is coming back or spreading. Ga 68 PSMA-11 is an injection given by a health care provider in the vein (intravenous) in preparation for an imaging test that can help detect cancer (called positron emission tomography or PET scan imaging). PET imaging done after Ga 68 PSMA-11 injection showed sites of prostate-specific membrane antigen (PSMA) positive lesions in men with prostate cancer.
Drug Name: Danyelza
Active Ingredient: naxitamab-gqgk
Approval Date: 11/25/2020
FDA-approved use: To treat high-risk refractory or relapsed neuroblastoma
Drug Use: DANYELZA is a drug used in combination with a medicine called granulocyte-macrophage colony-stimulating factor (GM-CSF) to treat children 1 year of age and older and adults with high-risk neuroblastoma in bone or bone marrow whose tumor – did not respond to or has come back after previous treatments and, has shown a partial response, minor response, or stable disease to prior therapy. DANYELZA is an injection given by a healthcare professional directly into a vein (intravenous infusion) on Days 1, 3 and 5 of each treatment cycle. GM-CSF is given under the skin (subcutaneously) daily starting 5 days prior to DANYELZA and continuing until the last day of DANYELZA administration of each cycle. In one trial, approximately 45 percent of patients receiving DANYELZA with GM-CSF experienced complete or partial shrinkage of their cancer; for 30% of those patients, shrinkage lasted 6 months or longer.. In another trial, 34% patients receiving DANYELZA with GM-CSF experienced complete or partial shrinkage of their cancer; for 23% of those patients, shrinkage lasted 6 months or longer for. DANYELZA was approved under FDA’s accelerated approval program, which provides earlier patient access to a promising new drug while the company continues to conduct clinical trials to confirm that the drug works well.
Drug Name: Imcivree
Active Ingredient: setmelanotide
Approval Date: 11/25/2020
FDA-approved use: To treat obesity and the control of hunger associated with pro-opiomelanocortin deficiency, a rare disorder that causes severe obesity that begins at an early age
Drug Use: IMCIVREE is a drug used for long-lasting weight management in patients 6 years and older who are obese because of a specific enzyme deficiency [(proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency)]. IMCIVREE is an injection given under the skin (subcutaneously) once daily. The daily dose is based on patient’s weight. After one-year treatment with IMCIVREE, 80% of patients with POMC or PCSK1 deficiency and 46% of patients with LEPR deficiency achieved at least 10% weight loss.
Drug Name: Oxlumo
Active Ingredient: lumasiran
Approval Date: 11/23/2020
FDA-approved use: To treat hyperoxaluria type 1
Drug Use: OXLUMO is a drug used to lower the level of urine oxalate in children and adults with primary hyperoxaluria type 1 (PH1). OXLUMO is an injection given under the skin (subcutaneously) once a month for the first three months. Some patients can then switch to an injection once every three months depending on their weight. In one trial, patients treated with OXLUMO had a 65% decrease in urinary oxalate after 6 months of treatment compared with a 12% decrease in patients who received placebo. In a second trial, patients treated with OXLUMO had a decrease in urinary oxalate of 71%.
Drug Name: Zokinvy
Active Ingredient: lonafarnib
Approval Date: 11/20/2020
FDA-approved use: To treat rare conditions related to premature aging
Drug Use: ZOKINVY is a drug used in patients one year of age and older with a certain body surface area to lower the risk of death in Hutchinson-Gilford Progeria Syndrome (HGPS), or, to treat certain processing-deficient progeroid laminopathies. ZOKINVY is a capsule taken by mouth twice daily. The total daily dose is based on the patient’s size (body surface area). ZOKINVY lowered the risk of death in patients with HGPS. The lifespan of HGPS patients treated with ZOKINVY increased by an average of three months through the first three years of follow-up and by an average of 2.5 years through the maximum follow-up time (11 years) compared to untreated patients.
Drug Name: Veklury
Active Ingredient: remdesivir
Approval Date: 10/22/2020
FDA-approved use: To treat COVID-19
Drug Use: VEKLURY is for use in adult and pediatric patients 12 years and older weighing at least 40 kg (88 lbs) for the treatment of coronavirus disease 2019 (COVID-19) requiring hospitalization. VEKLURY is an injection. It is given by a healthcare provider directly into the vein (intravenous infusion) once a day for 5 to 10 days. In one trial in patients with mild, moderate and severe COVID-19, the time to recovery was shorter in patients who received VEKLURY than in patients who received placebo. In a different trial in patients with moderate COVID-19, treatment with VEKLURY was better in improving patients’ condition in comparison to standard of care.
Drug Name: Inmazeb
Active Ingredient: atoltivimab, maftivimab, and odesivimab-ebgn
Approval Date: 10/14/2020
FDA-approved use: To treat ebola virus
Drug Use: INMAZEB is a drug used to treat pediatric and adult patients who have an infection caused by Zaire ebolavirus. INMAZEB is an injection. It is given one time by a healthcare provider directly into the vein (intravenous infusion). The amount of the drug to be administered is based on the patient’s weight. INMAZEB lowered the risk of dying from the infection. Out of 154 patients treated with INMAZEB, 52 patients (34%) died within 28-days in comparison to 78 out the 153 patients (51%) who were treated with another experimental drug.
Drug Name: Gavreto
Active Ingredient: pralsetinib
Approval Date: 09/04/2020
FDA-approved use: To treat non-small lung cancer
Drug Use: GAVRETO is a drug used to treat adult patients with non-small cell lung cancer (NSCLC) which has spread to other parts of the body (metastatic) and is caused by abnormal RET (rearranged during transfection) genes. GAVRETO is a capsule. Four capsules (400 mg) are taken by mouth once a day on an empty stomach. Fifty-seven percent of 87 patients with previously treated NSCLC experienced complete or partial shrinkage of their tumors. Shrinkage lasted more than 6 months for 80% of them. Out of 27 patients who had never undergone treatment, 70% experienced complete or partial shrinkage of their tumors. Shrinkage lasted more than 6 months for 58% of them. GAVRETO was approved under FDA’s accelerated approval program.
Drug Name: Detectnet
Active Ingredient: copper Cu 64 dotatate injection
Approval Date: 09/03/2020
FDA-approved use: To help detect certain types of neuroendocrine tumors
Drug Use: DETECTNET is a drug for detection of the specific type of tumors called somatostatin receptor positive neuro-endocrine tumors (NETs) in adults. DETECTNET is injected into a vein in preparation for an imaging test (called positron emission tomography scan or PET scan) to help detect the tumor.
Drug Name: Sogroya
Active Ingredient: somapacitan-beco
Approval Date: 08/28/2020
FDA-approved use: Growth hormone
Drug Use: SOGROYA is a drug for replacement of growth hormone in adults with growth hormone deficiency (GHD). SOGROYA is an injection given under the skin (subcutaneously) once every week. At the end of the trial, patients with GHD who were treated with SOGROYA experienced decrease of truncal fat percentage in comparison to placebo-treated patients who experienced increase in truncal fat percentage.
Drug Name: Winlevi
Active Ingredient: clascoterone
Approval Date: 08/26/2020
FDA-approved use: To treat acne
Drug Use: WINLEVI is used on the skin to treat acne vulgaris in patients 12 years and older. WINLEVI is a cream. It should be applied to the affected skin area twice a day. More patients achieved a reduction in the number of acne and clear, or almost clear, skin after 12 weeks of treatment with WINLEVI in comparison to those who were treated with vehicle cream (placebo).
Drug Name: Enspryng
Active Ingredient: satralizumab-mwge
Approval Date: 08/14/2020
FDA-approved use: To treat neuromyelitis optica spectrum disorder
Drug Use: ENSPRYNG is used to treat neuromyelitis optica spectrum disorder (NMOSD) in adult patients with a particular antibody (who are anti-aquaporin-4 or AQP4 antibody positive). ENSPRYNG is an injection given under the skin (subcutaneously). The first three injections are given two weeks apart followed by one injection every four weeks. ENSPRYNG reduces attacks of the disease. In Trial 1, 76% of ENSPRYNG-treated patients were relapse-free at 96 weeks, compared to 41% placebo-treated patients. In Trial 2, 91% of ENSPRYNG – treated patients were relapse-free at 96 weeks, compared to 57% placebo-treated patients.
Drug Name: Viltepso
Active Ingredient: viltolarsen
Approval Date: 08/12/2020
FDA-approved use: To treat Duchenne muscular dystrophy
Drug Use: VILTEPSO is a drug for the treatment of a particular type of Duchenne muscular dystrophy (DMD). It is to be used only in patients who have a specific mutation of the dystrophin gene. VILTEPSO is given by a health care professional once every week directly into the bloodstream through a needle in the vein. VILTEPSO increased levels of dystrophin in the muscles of treated patients. It is believed that this increase may predict clinical benefit in patients. VILTEPSO was approved under FDA’s accelerated approval program.
Drug Name: Olinvyk
Active Ingredient: oliceridine
Approval Date: 08/07/2020
FDA-approved use: To manage acute pain in certain adults
Drug Use: OLINVYK is a drug for the treatment of acute pain in adults when the pain is severe enough to require an intravenous opioid. OLINVYK is an injection. It is administered by a healthcare provider directly into the vein up to 27 milligrams per day. OLINVYK reduces post-surgical pain. It is not available for home use.
Drug Name: Evrysdi
Active Ingredient: risdiplam
Approval Date: 08/07/2020
FDA-approved use: To treat spinal muscular atrophy
Drug Use: EVRYSDI is a drug for the treatment of spinal muscular atrophy (SMA) in patients 2 months of age and older. EVRYSDI is taken as a liquid by mouth once a day after a meal. The amount of liquid is determined by the patient’s weight. In one trial, 41% (7/17) of treated infants were able to sit without support for more than 5 seconds after 12 months of treatment with EVRYSDI. In the second trial, patients treated with EVRYSDI, on average, improved muscle function after 12 months of treatment, while patients treated with placebo, on average, continued to deteriorate.
Drug Name: Lampit
Active Ingredient: nifurtimox
Approval Date: 08/06/2020
FDA-approved use: To treat Chagas disease in certain pediatric patients younger than age 18
Drug Use: LAMPIT is a drug used to treat Chagas disease in children less than 18 years of age. LAMPIT is a tablet. It is taken by mouth three times a day with food for 60 days. Total daily dose is determined by the child’s weight. The benefit of LAMPIT was measured by the number of patients who had a change in the antibodies to T. cruzi parasite (called serologic response). In the trial, 32% of patients who received LAMPIT for 60 days had serologic response after one year. LAMPIT was approved under FDA’s accelerated approval program.
Drug Name: Blenrep
Active Ingredient: belantamab mafodotin-blmf
Approval Date: 08/05/2020
FDA-approved use: To treat multiple myeloma
Drug Use: BLENREP is a drug used to treat a form of blood cancer called multiple myeloma. It is to be used in patients with whose cancer came back after, or did not respond to, at least four previous treatments. BLENREP is given directly into the vein (an I.V. infusion) by a heathcare professional every three weeks. It takes about 30 minutes to receive the BLENREP infusion. In the trial, 30 of 97 patients (31%) treated with BLENREP experienced an improvement in their disease. For seventy-three percent of responders that improvement lasted at least 6 months. BLENREP was approved under FDA’s accelerated approval program.
Drug Name: Monjuvi
Active Ingredient: tafasitamab-cxix
Approval Date: 07/31/2020
FDA-approved use: To treat relapsed or refractory diffuse large B-cell lymphoma
Drug Use: MONJUVI is used to treat adults with diffuse large B-cell lymphoma (DLBCL) whose disease has come back or has not improved after previous treatments. It is to be used when patients cannot receive a stem cell transplant. MONJUVI is given by a healthcare provider directly into the bloodstream through a needle in the vein. MONJUVI is given according to a specific cycle schedule and together with lenalidomide (a type of chemotherapy). In a clinical trial, of 71 patients with DLBCL assigned to get MONJUVI plus lenalidomide, 55% had complete or partial shrinkage of their tumors that lasted about 22 months. MONJUVI was approved under FDA’s accelerated approval program.
Drug Name: Xeglyze
Active Ingredient: abametapir
Approval Date: 07/24/2020
FDA-approved use: To treat head lice
Drug Use: XEGLYZE is a drug used to treat head lice in patients 6 months of age and older. XEGLYZE is a lotion applied once to dry hair. About 80% of patients treated with XEGLYZE were free of live lice in comparison to about 50% of patients who were treated with placebo.
Drug Name: Inqovi
Active Ingredient: decitabine and cedazuridine
Approval Date: 07/07/2020
FDA-approved use: To treat adult patients with myelodysplastic syndromes
Drug Use: INQOVI is used to treat adults with myelodysplastic syndromes (MDS) including chronic myelomonocytic leukemia. INQOVI is a tablet taken once a day on Days 1 through 5 of each 28 day cycle. In Trial 1, 18% of patients treated with INQOVI experienced complete response (specific improvements in bone marrow and blood cells) that lasted about 9 months. In Trial 2, 21% of patients treated with INQOVI, experienced complete response that lasted about 7.5 months. Additionally, about half of the patients who were formerly dependent on transfusions were able to no longer require transfusions during an 8-week period.
Drug Name: Rukobia
Active Ingredient: fostemsavir
Approval Date: 07/02/2020
FDA-approved use: To treat HIV
Drug Use: RUKOBIA is a drug for the treatment of human immunodeficiency virus-1 (HIV-1) infection in adults who have received several anti-HIV-1 regimens in the past, and, have HIV-1 virus that is resistant to many antiretroviral medicines, and, who are failing their current antiretroviral therapy because it is not effective, have intolerable side effects or other safety problems. RUKOBIA is a tablet that is taken by mouth twice a day in combination with other drugs approved for the treatment of HIV-1 infection. RUKOBIA reduced viral load of HIV-1.
Drug Name: Byfavo
Active Ingredient: remimazolam
Approval Date: 07/02/2020
FDA-approved use: For sedation
Drug Use: BYFAVO is a drug used to start and maintain sedation in adults undergoing short (less than 30 minutes) procedures. BYFAVO is given as an injection through a vein (intravenous) by a healthcare provider. BYFAVO provides sedation in adult patients undergoing short procedures.
Drug Name: Dojolvi
Active Ingredient: triheptanoin
Approval Date: 06/30/2020
FDA-approved use: To treat molecularly long-chain fatty acid oxidation disorders
Drug Use: DOJOLVI is a drug that provides calories and fatty acids for pediatric and adult patients with long-chain fatty acid oxidation disorder. DOJOLVI is a liquid mixed with meals or snacks four or more times per day. After four months of treatment, patients who received DOJOLVI showed similar heart function as patients who received trioctanoin (a different source of calories and fatty acids).
Drug Name: Zepzelca
Active Ingredient: lurbinectedin
Approval Date: 06/15/2020
FDA-approved use: To treat metastatic small cell lung cancer
Drug Use: ZEPZELCA is a drug used to treat adult patients with small cell lung cancer (SCLC) which has spread to other parts of the body (metastatic) on or after treatment with platinum-containing therapy. ZEPZELCA is given by a healthcare provider direcly into the vein (intravenous infusion) every three weeks. It takes about one hour to receive an infusion. Thirty seven (35%) of 105 patients with SCLC who received ZEPZELCA experienced partial shrinkage of their tumors. Tumor shrinkage lasted more than 6 months for 35% of those patients. ZEPZELCA was approved under FDA’s accelerated approval program.
Drug Name: Uplizna
Active Ingredient: inebilizumab-cdon
Approval Date: 06/11/2020
FDA-approved use: To treat neuromyelitis optica spectrum disorder
Drug Use: UPLIZNA is used to treat neuromyelitis optica spectrum disorder (NMOSD) in adult patients with a particular antibody (who are anti-aquaporin-4 or AQP4 antibody positive). UPLIZNA is given directly into the vein (intravenous infusion) over 90 minutes by a healthcare provider. First two infusions are given two weeks apart followed by one infusion every six months. UPLIZNA reduces attacks of the disease. The risk of an NMOSD relapse in the patients who were treated with UPLIZNA was reduced by 77% when compared to the placebo treatment group.
Drug Name: Tauvid
Active Ingredient: flortaucipir F18
Approval Date: 05/28/2020
FDA-approved use: Diagnostic agent for patients with Alzheimer’s disease
Drug Use: TAUVID is a drug for the visual detection of aggregated neurofibrillary tangles or NFTs in the brain of adult patients with suspected Alzheimer’s disease (AD). TAUVID is injected into a vein by a healthcare provider in preparation for an imaging test (called positron emission tomography scan or PET scan) to detect the NFTs. TAUVID detects NFTs on PET scan images of the brain.
Drug Name: Artesunate
Active Ingredient: artesunate
Approval Date: 05/26/2020
FDA-approved use: To treat severe malaria
Drug Use: ARTESUNATE is a drug used for initial treatment of severe malaria in children and adults. Treatment of severe malaria with artesunate should always be followed by a complete treatment course of an appropriate antimalarial drug. ARTESUNATE is an injection given directly into the vein by a healthcare professional. First three doses are given during the first 24 hours of treatment (at 0, 12 and 24 hours) followed by once daily administration until patient is able to take oral antimalarial therapy. The number of patients treated with ARTESUNATE who died in the hospital (called in-hospital mortality rate) was lower than the number who died in the control group treated with quinine (a drug approved for the treatment of acute malaria). The in-hospital mortality rate was 13% for ARTESUNATE-treated patients versus 21% for quinine-treated patients.
Drug Name: Cerianna
Active Ingredient: fluoroestradiol F18
Approval Date: 05/20/2020
FDA-approved use: Diagnostic imaging agent for certain patients with breast cancer
Drug Use: CERIANNA is a drug for the visual detection of estrogen receptor (ER)-positive lesions in addition to tissue biopsy in patients with recurrent or metastatic breast cancer. CERIANNA is injected into a vein by a healthcare provider in preparation for an imaging test (called positron emission tomography scan or PET scan) to detect the lesions. CERIANNA is to be used in addition to a tissue biopsy. CERIANNA detects ER-positive lesions on PET scan images of patients with breast cancer.
Drug Name: Qinlock
Active Ingredient: ripretinib
Approval Date: 05/15/2020
FDA-approved use: To treat advanced gastrointestinal-stromal tumors
Drug Use: QINLOCK is drug used to treat adult patients with gastrointestinal stromal tumor (GIST) whose disease: cannot be surgically removed or, has spread throughout the body (metastatic GIST), and has been treated with at least three prior treatments. QINLOCK is a tablet taken once daily. The benefit of QINLOCK was evaluated by measuring the length of time tumors did not grow after treatment (progression-free survival or PFS). The progression-free survival for patients taking QINLOCK was about 6 months compared to 1 month for patients taking a placebo.
Drug Name: Retevmo
Active Ingredient: selpercatinib
Approval Date: 05/08/2020
FDA-approved use: To treat lung and thyroid cancers
Drug Use: RETEVMO is a drug used to treat certain cancers caused by abnormal RET (rearranged during transfection) genes in: adult patients with non-small cell lung cancer (NSCLC) which has spread to other parts of the body (metastatic), adults and children 12 years of age and older with advanced or metastatic medullary thyroid cancer (MTC), adults and children 12 years of age and older with advanced or metastatic thyroid cancer who have received radioactive iodine that did not work or is no longer working. RETEVMO is a capsule usually taken by mouth twice a day. Sixty-four percent of the 105 patients with previously treated NSCLC, experienced complete or partial shrinkage of their tumors which lasted more than 6 months for 81% of them. Out of 39 patients who had never undergone treatment, 84% experienced complete or partial shrinkage of their tumors which lasted more than 6 months for 58% of them. Sixty-nine percent of the 55 previously treated patients for MTC experienced complete or partial shrinkage of their tumors which lasted more than 6 months for 76% of them. Out of 88 patients who had never undergone treatment with an approved drug, 73% experienced complete or partial shrinkage of their tumors which lasted more than 6 months for 61% of them. Seventy-nine percent of the 19 previously treated patients with thyroid cancer experienced complete or partial shrinkage of their tumors which lasted more than 6 months for 87% of them. All eight patients who had not received therapy other than radioactive iodine therapy experienced complete or partial shrinkage of their tumors which lasted more than 6 months for 75 % of them. RETEVMO was approved under FDA’s accelerated approval program.
Drug Name: Tabrecta
Active Ingredient: capmatinib
Approval Date: 05/06/2020
FDA-approved use: To treat patients with non small cell lung cancer
Drug Use: TABRECTA is a drug used to treat adult patients with a type of non-small cell lung cancer (NSCLC) which: has a specific gene mutation (mesenchymal epithelial transition or MET mutation) and, has spread to other parts of the body (metastatic). TABRECTA is a capsule taken by mouth twice a day. Nineteen (68%) of 28 patients with NSCLC who have not been previously treated and who received TABRECTA experienced complete or partial shrinkage of their tumors. Tumor shrinkage lasted more than 12 months for 47% of those patients. Twenty-eight (41%) of 69 patients with NSCLC who have been previously treated and who received TABRECTA experienced partial shrinkage of their tumors. Tumor shrinkage lasted more than 12 months for 32% of those patients. TABRECTA was approved under FDA’s accelerated approval program.
Drug Name: Ongentys
Active Ingredient: opicapone
Approval Date: 04/24/2020
FDA-approved use: To treat patients with Parkinson’s disease experiencing “off” episodes
Drug Use: ONGENTYS is a drug used to treat patients with Parkinson’s disease (PD) who are having “off” episodes while taking drugs (levodopa and carbidopa) for the treatment of Parkinson’s disease. An “off” episode is a time when a patient’s medications are not working well, leading to an increase in Parkinson’s symptoms, such as tremor and difficulty walking. ONGENTYS is a capsule taken by mouth once daily at bedtime, in addition to drugs for the treatment of Parkinson’s disease (levodopa and carbidopa). Patients treated with ONGENTYS had decreased “off” time compared to patients treated with placebo.
Drug Name: Trodelvy
Active Ingredient: sacituzumab govitecan-hziy
Approval Date: 04/22/2020
FDA-approved use: To treat adult patients with metastatic triple-negative breast cancer who received at least two prior therapies for metastatic disease
Drug Use: TRODELVY is a drug for the treatment of adults with triple-negative breast cancer that has spread to other parts of the body (metastatic). It should be used in patients who have received at least two prior therapies for their metastatic disease. TRODELVY is an injection. It is given directly into the vein (intravenous infusion) by a healthcare provider once weekly on Days 1 and 8 of 21-day treatment cycles. The trial measured the percent of patients whose tumors completely or partially shrank after treatment (objective response rate). In the trial, approximately 30 percent of 108 patients taking TRODELVY experienced complete or partial shrinkage of their tumors that lasted about 8 months. TRODELVY was approved under FDA’s accelerated approval program.
Drug Name: Pemazyre
Active Ingredient: pemigatinib
Approval Date: 04/17/2020
FDA-approved use: To treat certain patients with cholangiocarcinoma, a rare form of cancer that forms in bile ducts
Drug Use: PEMAZYRE is a drug used for treatment of adults with bile duct cancer (cholangiocarcinoma) that has spread to other parts of the body (metastatic) or cannot not be removed by surgery. It should be used in patients who have been previously treated with chemotherapy and whose cancer has a certain type of abnormality in the FGFR2 gene. PEMAZYRE is a tablet taken by mouth once a day for 14 days followed by 7 days off treatment to complete a 21-day treatment cycle. In the trial, 38 of 107 patients (36%) treated with PEMAZYRE achieved partial or complete shrinkage of the tumor (overall response rate). Of these patients, 63% had a cancer shrinkage lasting 6 months or longer and 18% had it lasting 12 months or longer. PEMAZYRE was approved under FDA’s accelerated approval program.
Drug Name: Tukysa
Active Ingredient: tucatinib
Approval Date: 04/17/2020
FDA-approved use: To treat advanced unresectable or metastatic HER2-positive breast cancer
Drug Use: TUKYSA is a drug for treatment of adults with human epidermal growth factor receptor (HER)2-positive breast cancer that has spread to other parts of the body including the brain (metastatic) or cannot not be removed by surgery. It should be used in patients who have been previously treated for their metastatic disease with at least one anti-HER2 regimen and in combination with two other medications for the treatment of metastatic breast cancer (trastuzumab and capecitabine). TUKYSA is a tablet taken by mouth two times a day in combination with trastuzumab and capecitabine. The trial measured the length of time tumors did not grow after treatment (progression-free survival or PFS). The progression-free survival for patients taking TUKYSA together with trastuzumab and capecitabine was about 8 months compared to 6 months for patients taking a placebo with trastuzumab and capecitabine.
Drug Name: Koselugo
Active Ingredient: selumetinib
Approval Date: 04/10/2020
FDA-approved use: To treat neurofibromatosis type 1, a genetic disorder of the nervous system causing tumors to grow on nerves
Drug Use: KOSELUGO is used to to treat children 2 years of age and older with a type of tumor called plexiform neurofibroma or PN that occurs in a rare disease called neurofibromatosis type 1 (NF1). KOSELUGO is used in patients whose tumors cause symptoms and could not be surgically removed. KOSELUGO is a capsule taken two times a day. In the trial, 33 of 50 patients (66%) who received KOSELUGO experienced partial tumor shrinkage. Of these patients, 82% had a tumor shrinkage lasting 12 months or longer.
Drug Name: Zeposia
Active Ingredient: ozanimod
Approval Date: 03/25/2020
FDA-approved use: To treat relapsing forms of multiple sclerosis
Drug Use: ZEPOSIA is a drug used for the treatment of adults with relapsing forms of multiple sclerosis (RMS) including: clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive disease. ZEPOSIA is a capsule taken by mouth once daily. ZEPOSIA is initially started at a low dose and then slowly increased over the first week. ZEPOSIA was better in reducing the risk of disease relapse in comparison to interferon β1a, a product approved for the treatment of relapsing forms of MS.
Drug Name: Isturisa
Active Ingredient: osilodrostat
Approval Date: 03/06/2020
FDA-approved use: To treat adults with Cushing’s disease who either cannot undergo pituitary gland surgery or have undergone the surgery but still have the disease
Drug Use: ISTURISA is a drug for the treatment of adults with Cushing’s disease: who cannot have pituitary gland surgery, or, who have had pituitary gland surgery, but the surgery did not cure their Cushing’s disease.
Drug Name: Sarclisa
Active Ingredient: isatuximab
Approval Date: 03/02/2020
FDA-approved use: To treat multiple myloma
Drug Use: SARCLISA is a drug used to treat a form of blood cancer called multiple myeloma. SARCLISA is for adult patients whose cancer returned or did not respond to, at least two previous treatments for multiple myeloma. It is to be used in combination with two other drugs: dexamethasone (a type of corticosteroid) and pomalidomide. SARCLISA is an injection. It is given by a healthcare profesional directly into the vein (intravenous infusion) every week for 4 weeks followed by every 2 weeks. In the trial, patients who received SARCLISA (in combination with pomalidomadie and dexamethasone) lived longer without the cancer growing (average about 12 months) in comparison to patients who received pomalidomadie and dexamethasone combination only (average about 6 months).
Drug Name: Nurtec ODT
Active Ingredient: rimegepant
Approval Date: 02/27/2020
FDA-approved use: To treat migraine
Drug Use: NURTEC ODT is a drug used for treatment of acute migraine with or without aura in adults. NURTEC ODT is a tablet taken by mouth once a day, as needed. A higher percentage of patients did not have a headache two hours after taking NURTEC ODT compared to patients who took a placebo. Also, a higher percentage of patients who took NURTEC ODT did not have their most bothersome migraine-associated symptom (e.g., light sensitivity, nausea, or sound sensitivity) two hours after taking the drug, in comparison to patients who received placebo.
Drug Name: Barhemsys
Active Ingredient: amisulpride
Approval Date: 02/26/2020
FDA-approved use: To help prevent nausea and vomiting after surgery
Drug Use: BARHEMSYS is a drug used in adults to prevent or treat postoperative nausea and vomiting (PONV). It is to be used: for prevention of PONV either alone, or together with a different drug for PONV prevention, for treatment of PONV in patients who have not received any drug for the prevention of PONV, or those who have received a different drug for the prevention for PONV. BARHEMSYS is an injection given directly into the vein (intravenous infusion) by a healthcare professional over 1-2 minutes. Patients taking BARHEMSYS experienced less nausea/vomiting and less need for additional medication to prevent or treat PONV in comparison to patients receiving placebo.
Drug Name: Vyepti
Active Ingredient: eptinezumab-jjmr
Approval Date: 02/21/2020
FDA-approved use: For the preventive treatment of migraine in adults
Drug Use: VYEPTI is a drug used for the preventive treatment of migraine in adults. VYEPTI is an injection given directly into the vein (intravenous infusion) by a healthcare provider every three months. It takes about 30 minutes to receive the VYEPTI infusion. Over the three-month treatment period, patients treated with VYEPTI experienced fewer days of migraine headaches in comparison to patients who received the placebo treatment.
Drug Name: Nexletol
Active Ingredient: bempedoic acid
Approval Date: 02/21/2020
FDA-approved use: To treat adults with heterozygous familial hypercholesterolemia or established atherosclerotic cardiovascular disease who require additional lowering of LDL-C
Drug Use: NEXLETOL is a drug for the treatment of high LDL cholesterol, which is sometimes referred to as “bad cholesterol”. NEXLETOL is a tablet used once daily, in addition to low cholesterol diet and the highest dose of a statin (another drug commonly used to lower cholesterol) that a patient can tolerate. It should only be taken when LDL cholesterol needs to be lowered further. NEXLETOL lowers LDL cholesterol.
Drug Name: Pizensy
Active Ingredient: lactitol
Approval Date: 02/12/2020
FDA-approved use: To treat chronic idiopathic constipation (CIC) in adults
Drug Use: PIZENSY is used to treat a type of constipation called chronic idiopathic constipation (CIC) in adults. PIZENSY is a powder. The content of two packets or bottle caps should be mixed with a common beverage and taken once a day. The content of one packet or bottle cap can be taken once a day if the patient develops persistent loose stools with intake of two packets or bottle caps. More patients who received PIZENSY experienced an increase in the number of complete spontaneous bowel movements (CSBMs) to at least 3 bowel movements per week than those who received placebo.
Drug Name: Tazverik
Active Ingredient: tazemetostat
Approval Date: 01/23/2020
FDA-approved use: To treat epithelioid sarcoma
Drug Use: TAZVERIK is a drug used to treat a type of cancer called advanced epithelioid sarcoma. It is to be used in patients 16 years and older when cancer has spread and cannot be completely removed by surgery. TAZVERIK is a tablet. Four tablets (a total of 800 mg) are taken twice a day. Out of 62 patients who received TAZVERIK, 9 patients (15%) achieved partial or complete shrinkage of the cancer. For 6 of these patients (67%) the shrinkage lasted six months or longer. TAZVERIK was approved under FDA’s accelerated approval program.
Drug Name: Tepezza
Active Ingredient: teprotumumab-trbw
Approval Date: 01/21/2020
FDA-approved use: To treat Thyroid eye disease
Drug Use: TEPEZZA is a drug for the treatment of thyroid eye disease. TEPEZZA is an injection. It is given by a healthcare provider directly into the vein (an intravenous infusion) once every three weeks for a total of eight infusions. It takes about 60-90 minutes to receive the infusion. After 6 months of treatment, a higher proportion of patients treated with TEPEZZA (71-83%) demonstrated reduction in proptosis in comparison to patients treated with placebo (10-20%).
Drug Name: Ayvakit
Active Ingredient: avapritinib
Approval Date: 01/09/2020
FDA-approved use: To treat adults with unresectable or metastatic gastrointestinal stromal tumor (GIST)
Drug Use: AYVAKIT is used to treat adult patients with gastrointestinal stromal tumor (GIST) whose disease: is caused by certain abnormal platelet-derived growth factor receptor alpha (PDGFRA) genes and, cannot be surgically removed or, has spread throughout the body (metastatic GIST). AYVAKIT is a tablet taken once daily on an empty stomach. Thirty-six out of 43 patients (84%) experienced complete or partial shrinkage of the tumor which lasted six months or longer in 22 patients (61%).
