Washington, DC – The 2023 US Food and Drug Administration approval of omaveloxolone (Skyclarys®) for the treatment of Friedreich ataxia (FA) in patients aged 16 or more years was a significant step forward for the FA community. Omaveloxolone promotes improved mitochondrial function and boosts antioxidant activity—but it does not offer...
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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...
The U.S. Food and Drug Administration (FDA) today approved an imaging drug known as Cytalux (pafolacianine), which is attracted to ovarian cancer tissue and illuminates it when exposed to fluorescent light, allowing surgeons to more easily find and more precisely remove the cancer. Physicians at the Center for Precision Surgery in the Abramson Cancer Center...
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...
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...