BioSpace News Archive

UPLIFT, a single-arm registration strategy evaluating upifitamab rilsodotin (UpRi) in platinum-resistant ovarian cancer, on track to begin in Q1 2021

Company granted 180 days of exclusivity under FDA Competitive Generic Therapy (CGT) approval pathway

Pfizer Inc. (NYSE: PFE) and OPKO Health Inc. (NASDAQ: OPK) announced today that the European Medicines Agency (EMA) has validated for review the Marketing Authorization Application (MAA) for somatrogon, a long-acting recombinant human growth hormone that is intended to be administered once-weekly for the treatment of pediatric patients with growth hormone deficiency (GHD)

PTC Therapeutics, Inc., announced that the European Medicines Agency Committee for Medicinal Products for Human Use has recommended the approval of Evrysdi™ for the treatment of 5q spinal muscular atrophy in patients 2 months and older, with a clinical diagnosis of SMA Type 1, Type 2 or Type 3 with one to four SMN2 copies.

Fulcrum Therapeutics, Inc. (Nasdaq: FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced the company’s recognition of Rare Disease Day 2021.

If approved, the vaccine would help protect adults against 20 serotypes responsible for the majority of invasive pneumococcal disease and pneumonia

Ipsen Receives Positive CHMP Opinion Recommending Cabometyx ® in Combination With Opdivo ® as First-line Treatment for Patients Living With Advanced Renal Cell Carcinoma

Fulcrum Therapeutics, Inc. (Nasdaq: FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that its fourth quarter and full year 2020 financial results will be released on Thursday, March 4, 2021 before the U.S. financial markets open.

– In Partnership with the Sickle Cell Community Consortium, Agios Works to Address Educational Resource Gaps Caused by COVID-19 for Students with Sickle Cell Disease – – Company to Host Facebook Live Discussion with Pyruvate Kinase (PK) Deficiency Patient, Caregiver and Key Opinion Leader Today at 2 p.m. ET –

Ascendis Pharma A/S (Nasdaq: ASND), a biopharmaceutical company that utilizes its innovative TransCon technologies to address unmet medical needs, today announced the launch of Outgrow GHD , a new online educational resource designed to help caregivers of children with growth hormone deficiency (GHD) in the United States (U.S.) navigate their personal GHD journeys.