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...
treatment News
WALTHAM, Mass. — Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) today announced that the U.S. Food and Drug Administration (FDA) has approved EMPAVELI® (pegcetacoplan) as the first treatment for C3 glomerulopathy (C3G) or primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) in patients 12 years of age and older, to reduce proteinuria. C3G and primary...
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)....
INGELHEIM, Germany – The FDA has approved nerandomilast (Jascayd; Boehringer Ingelheim) tablets to treat idiopathic pulmonary fibrosis (IPF)—a rare, progressive, and severe disease that does not have a cure and has limited treatment—marking this therapy as the first approved treatment for this population in over 10 years.1 Understanding Idiopathic...
BRIDGEWATER, N.J. — Insmed Incorporated (Nasdaq: INSM), a people-first global biopharmaceutical company striving to deliver first- and best-in-class therapies to transform the lives of patients facing serious diseases, today announced that the U.S. Food and Drug Administration (FDA) has approved first-in-class BRINSUPRI™ (brensocatib 10 mg and 25 mg tablets), an oral, once-daily treatment...
ANDEMBRY inhibits the top of the HAE cascade by targeting factor XIIa and provides sustained protection from attacks Once-monthly dosing reduced HAE attacks by a median of more than 99 percent and a least squares mean of 89.2 percent, compared to placebo ANDEMBRY approval expands CSL’s HAE franchise and underscores...
WALTHAM, Mass. — Sobi North America, the North American affiliate of Swedish Orphan Biovitrum AB (Sobi®), today announced that the U.S. Food and Drug Administration (FDA) approved Doptelet® (avatrombopag) for the treatment of thrombocytopenia in pediatric patients one year and older with persistent or chronic immune thrombocytopenia (ITP) who have had an...
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....