FDA has approved Evkeeza (evinacumab-dgnb) injection as an add-on treatment for patients aged 12 years and older with homozygous familial hypercholesterolemia (HoFH), a genetic condition that causes severely high cholesterol. HoFH is a rare, life-threatening condition that occurs in approximately 1 in 250,000 individuals. Patients with HoFH have two mutations in a...
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CAMBRIDGE, Mass. – Blueprint Medicines Corporation (NASDAQ: BPMC) today announced that the U.S. Food and Drug Administration (FDA) has approved AYVAKIT™ (avapritinib) for the treatment of adult patients with advanced systemic mastocytosis (Advanced SM), including aggressive SM (ASM), SM with an associated hematological neoplasm (SM-AHN) and mast cell leukemia (MCL)....
FOSTER CITY, Calif. — Gilead Sciences, Inc., (Nasdaq: GILD) today announced the U.S. Food and Drug Administration (FDA) approved a new, expanded indication for Biktarvy® (bictegravir 50 mg/emtricitabine 200 mg/tenofovir alafenamide 25 mg tablets, B/F/TAF) to treat people with HIV (PWH) who have suppressed viral loads with known or suspected...
Paris, France – The FDA has approved Ipsen’s Sohonos (palovarotene) to reduce new abnormal bone formation in people living with fibrodysplasia ossificans progressiva (FOP), an ultra-rare genetic disease that transforms the body’s soft issue into bone. Sohonos is indicated to treat girls 8 years of age and older and boys...
SILVER SPRING, Md. – Today, the U.S. Food and Drug Administration approved Voxzogo (vosoritide) injection to improve growth in children five years of age and older with achondroplasia and open epiphyses (growth plates), meaning these children still have the potential to grow. Achondroplasia is the most common form of dwarfism....
SILVER SPRING, Md. — Today, the U.S. Food and Drug Administration approved Ogsiveo (nirogacestat) tablets for adult patients with progressing desmoid tumors who require systemic treatment. Ogsiveo is the first drug to be approved for the treatment of patients with desmoid tumors, a rare subtype of soft tissue sarcomas. Desmoid tumors...
SILVER SPRING, Md. – Today, the U.S. Food and Drug Administration approved Nulibry (fosdenopterin) for injection to reduce the risk of death due to Molybdenum Cofactor Deficiency Type A, a rare, genetic, metabolic disorder that typically presents in the first few days of life, causing intractable seizures, brain injury and...
SILVER SPRING, Md.— Today, the U.S. Food and Drug Administration approved Amtagvi, the first cellular therapy indicated for the treatment of adult patients with a type of skin cancer (melanoma) that is unable to be removed with surgery (unresectable) or has spread to other parts of the body (metastatic) that...
PARIS, France – Ipsen announced today that the U.S. Food and Drug Administration (FDA) has approved the supplemental new drug application for Onivyde® (irinotecan liposome injection) plus oxaliplatin, fluorouracil and leucovorin (NALIRIFOX) as a first-line treatment in adults living with metastatic pancreatic adenocarcinoma (mPDAC). This is the second approval for...
TARRYTOWN, N.Y. and PARIS – Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) and Sanofi today announced that the U.S. Food and Drug Administration (FDA) has approved the PD-1 inhibitor Libtayo® (cemiplimab-rwlc) for the first-line treatment of patients with advanced non-small cell lung cancer (NSCLC) whose tumors have high PD-L1 expression (tumor proportion score ≥50%), as determined...