Asparlas from Servier Pharmaceuticals is an asparagine-specific enzyme that is FDA-indicated as a treatment for acute lymphoblastic leukemia in patients aged one month to 21 years. In 2018, FDA approved the drug for use as part of a multi-agent chemotherapeutic regimen for acute lymphoblastic leukemia (ALL).

Gamifant from Sobi, Inc. is an interferon gamma (IFNγ) blocking antibody that is FDA-indication to treat adult and pediatric patients with primary hemophagocytic lymphohistiocytosis (HLH). The indication covers refractory, recurrent or progressive disease or intolerance with conventional HLH therapy. First approved in the U.S. in 2018, Gamifant has run into regulatory roadblocks in Europe. The European…

Tukysa from Seagen won FDA approval for HER2-positive breast cancer in conjunction with chemotherapy (trastuzumab and capecitabine) to treat advanced unresectable or metastatic HER2-positive breast cancer. The indication covers patients with brain metastases who previously received anti-HER2-based therapies for metastatic cancer.

In 2018, FDA indicated Annovera from TherapeuticsMD to prevent pregnancy. A drug-device combination, Annovera combines a ring-shaped vaginal delivery system with hormones. The manufacturer designed the device to remain in place for three weeks followed by removal for one week. The product is effective for up to one year (or 13 menstrual cycles). The manufacturer…

Lorbrena from Pfizer was initially FDA indicated to treat adults with metastatic non-small cell lung cancer (NSCLC) whose tumors are anaplastic lymphoma kinase (ALK)-positive based an FDA-approved test. In March, Pfizer announced that the drug had won an expanded indication for use as a first-line therapy for patients with anaplastic lymphoma kinase (ALK)-positive non-small cell…

Braftovi Mektovi from Array BioPharma won FDA approval in 2018 for patients with unresectable or metastatic melanoma with a BRAF V600E or V600K mutation. Pfizer acquired Array BioPharma in 2019. Braftovi Mektovi is an example of a BRAF/MEK inhibitor combination. The BRAF gene supports chemical signaling between the cell’s nuclear and the cell’s exterior. The MEK…

Piqray from Novartis is FDA indicated to treat hormone receptor (HR)- positive, human epidermal growth factor receptor 2 (HER2)-negative, PIK3CA-mutated, advanced or metastatic breast cancer. The FDA indication requires the use of an FDA-approved test following progression on or after an endocrine-based regimen.

Many researchers are exploring the potential of the psychedelic compound psilocybin to treat conditions such as depression and post-traumatic stress disorder. But psilocybin offers broader therapeutic promise as it appears to spur neuroplasticity, according to Greg McKee, CEO of Tryp Therapeutics (San Diego). The company is exploring the use of psilocybin-based drugs for treating eating disorders…

Orgovyx from Myovant Sciences is a gonadotropin-releasing hormone receptor antagonist that is FDA indicated to treat adults with advanced prostate cancer. FDA approved the drug in December 2020. In a press release, Richard Pazdur, FDA’s acting director of the Office of Oncologic Diseases, noted that ORGOVYX is “the first oral drug in this class and it…

FDA has indicated Merck‘s Recarbio for infections resulting from susceptible gram-negative microorganisms. The drug combines imipenem (a penem antibacterial), cilastatin (a renal dehydropeptidase inhibitor) and relebactam (a beta-lactamase inhibitor). Current FDA indications for the drug include hospital-acquired bacterial pneumonia and ventilator-associated bacterial pneumonia, complicated urinary tract infections and complicated intra-abdominal infections.

FDA has indicated Moxidectin from Medicines Development for Global Health to treat onchocerciasis (river blindness) resulting from Onchocerca volvulus in patients aged 12 years and older. The drug won that indication in June 2020. The drug is the first novel treatment for river blindness in two decades. The anthelmintic drug also has veterinary applications. It is…

FDA has indicated Karyopharm Therapeutics‘ nuclear export inhibitor Xpovio for relapsed or refractory multiple myeloma (RRMM). The indication covers its use in conjunction with the steroid dexamethasone. Patients receiving the drug should receive at least four previous therapies. Furthermore, the indication covers patients with myeloma that is refractory to at least two proteasome inhibitors, two immunomodulatory agents and…

Last year, FDA approved the celecoxib oral solution Elyxyb from Dr. Reddy’s Laboratories as an acute treatment of migraine with or without aura in adults. Formerly known as DFN-15, Elyxyb is a novel selective inhibitor of cyclooxygenase-2. The company received FDA approval for the drug in May 2020. The company formulated the drug as an oral solution…

Pemazyre (pemigatinib) from Innovent Biologics is a novel treatment for cholangiocarcinoma, a type of bile duct cancer. The indication covers unresectable locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or other rearrangement. Innovent continues to roll the drug out throughout the world. In June, it won regulatory approval in Taiwan…

In one sense, Veklury (remdesivir) is not a new drug. Gilead Sciences initially developed remdesivir (GS-5734) in reaction to the 2014 Ebola outbreak. The drug remained investigational, however, until the COVID-19 pandemic. In 2020, it became the first FDA-approved drug to treat COVID-19. Last year, it raked in $2.811 billion in revenue.

ROZLYTREK from Roche‘s Genentech division is FDA-indicated for adult patients with metastatic non-small cell lung cancer whose tumors are ROS1-positive. In addition, it is indicated for adults and children 12 and older with solid tumors. The latter indication requires that the tumors have a neurotrophic tyrosine receptor kinase gene fusion without a known acquired resistance mutation. Other requirements…

Nexletol is similar to Nexlizet in its FDA indication as an adjunct to diet and statin therapy to treat adults with heterozygous familial hypercholesterolemia or established atherosclerotic cardiovascular disease who need additional help lowering of LDL cholesterol. In September, Nexletol began a direct-to-consumer campaign advertising the potential of the drug to support LDL cholesterol-lowering as an adjunct…

Nexlizet from Esperion is FDA-indicated for heterozygous familial hypercholesterolemia or established atherosclerotic cardiovascular disease. The indication is intended as an adjunct to diet and statin therapy for patients who need additional help reducing LDL cholesterol levels. The drug, which combines two cholesterol-reducing medicines (bempedoic acid and ezetimibe), won FDA approval early last year.  

FDA has approved TAZVERIK from Epizyme for multiple indications. The first-in-class EZH2 inhibitor is now indicated for adults and pediatric patients aged 16 years and older with metastatic or locally advanced epithelioid sarcoma not eligible for complete resection. The drug is also indicated for adults with relapsed or refractory follicular lymphoma whose tumors are positive for…

Reyvow (lasmiditan) is an FDA-approved treatment from Lilly for the acute treatment of migraine with or without aura in adults. FDA has not approved it as a preventive treatment of migraine. In October, Lilly announced that the drug showed superior pain freedom after two hours of ingestion in two-thirds of migraine attacks in a Phase…

FDA has indicated Lilly‘s OLUMIANT (baricitinib) to treat adult patients with moderately to severely active rheumatoid arthritis. In particular, the agency has recommended the drug for patients with an inadequate response to one or more tumor necrosis factor (TNF) antagonist therapies. In June, Lilly and its partner Incyte announced that the drug appeared to reduce…

In November, 2020, Zokinvy (lonafarnib) from Eiger BioPharmaceuticals became the first drug to win FDA approval to lower the risk of death resulting from Hutchinson-Gilford progeria syndrome, a progressive genetic disorder that leads to rapid aging in children. The FDA indication also covered the treatment of some processing-deficient progeroid laminopathies in patients one year of age…

Qinlock (ripretinib) is indicated to treat adult patients with advanced gastrointestinal stromal tumors (GIST). The drug from Qinlock won FDA approval on May 15, 2020. In the Phase 3 INVICTUS study, the drug demonstrated clinical benefits with dose escalation for patients with advanced gastrointestinal stromal tumors with disease progression.      

FDA has indicated Gavreto (pralsetinib) from Blueprint Medicines to treat adult patients with metastatic RET fusion-positive non-small cell lung cancer (NSCLC). The drug was formerly known as BLU-667 and won FDA approval in September 2020. CStone, which is developing the drug in China, received approval from that country’s regulatory authority, making it the first novel therapy for…

Ayvakit from Blueprint Medicines Corp. was initially FDA-indicated to treat adults with unresectable or metastatic GIST harboring a platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation, including PDGFRA D842V mutations. In June, the drug won expanded use to include advanced systemic mastocytosis based on data from the single-arm, open-label EXPLORER and PATHFINDER clinical studies.…