A request for additional data may cause a 3-month delay for potential approval of BioMarin Pharmaceutical’s hemophilia A gene therapy.
Although the agency stated that Veru Inc’s experimental drug for COVID-19 met the main goal of reducing the death rate in a late-stage trial, it flagged a number of uncertainties with the data.
A patient taking part in an FDA-sanctioned clinical trial assessing a CRISPR-based therapy for a rare form of Duchenne muscular dystrophy (DMD) has died. Few details related to the circumstances of the patient’s death are known at this time, including whether or not he had been dosed with the one-time gene therapy.
Akebia Therapeutics has submitted a Formal Dispute Resolution Request with the FDA regarding the rejection of vadadustat in anemia due to chronic kidney disease.
Apellis is submitting additional 24-month efficacy data to supplement the NDA for its intravitreal candidate pegcetacoplan for GA secondary to AMD.
Minneapolis-based DiaMedica Therapeutics plans to conduct additional studies in order to resolve a clinical hold on its DM199 program for the treatment of acute ischemic stroke (AIS).
The therapy Tecvayli is a bispecific antibody, which is engineered to redirect body’s immune system to recognize and kill cancer cells. It is the first drug of its class to be approved for multiple myeloma.
The FDA’s Obstetrics Reproductive and Urologic Drugs Advisory Committee voted nearly unanimously to withdraw the approval of Covis’ preterm birth drug Makenna.
The authorization applies for people unable to get updated Omicron-tailored boosters, or those who would choose not to receive any other booster dose.
The FDA is likely to push back the Prescription Drug User Fee Act target action date for Travere Therapeutics’ sparsentan by three months.