On March 22, an FDA Advisory Committee (AdCom) unanimously voted in favor of accelerated approval for Biogen’s tofersen for a rare form of amyotrophic lateral sclerosis (ALS). Panelists voted 9-0 that tofersen’s demonstrated reduction in plasma neurofilament—a biomarker of neurodegeneration—was “reasonably likely” to predict clinical benefit in ALS. The AdCom...
treatment News
Washington DC – The FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee agreed that Vertex Pharmaceuticals had sufficiently demonstrated the safety of its investigational sickle cell disease gene-edited therapy exagamglogene autotemcel (exa-cel). Still, the panel of external experts did indicate that Vertex and its partner CRISPR Therapeutics could follow treated...
BOSTON, Mass. and SURESNES, France — Servier, a global leader in oncology focused on delivering meaningful therapeutic progress for the patients it serves, today announced the FDA filing acceptance and priority review for a New Drug Application (NDA) for vorasidenib, as well as the EMA granting accelerated assessment for the...
Recently completed medical research could lead to the first ever FDA approved drugs for FSGS and IgA Nephropathy—two conditions with dismal prognoses using current interventions. Nephrology Associates of Northern Illinois and Indiana (NANI) physicians and researchers nationwide collaborated on groundbreaking studies. IgA Nephropathy (IgAN) is an autoimmune disease that affects the kidneys...
EAST HANOVER, N.J. — Novartis announced that the U.S. Food and Drug Administration (FDA) approved Lutathera® (lutetium Lu 177 dotatate) for the treatment of pediatric patients 12 years and older with somatostatin receptor-positive (SSTR+) gastroenteropancreatic neuroendocrine tumors (GEP-NETs), including foregut, midgut, and hindgut NETs. This approval makes Lutathera the first therapy...
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...
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....