Aug 16 (Reuters) - The U.S. Food and Drug Administration (FDA) on Wednesday approved French drugmaker Ipsen's (IPN.PA) drug for a rare bone disorder, making it the first treatment available to patients with the condition that causes abnormal bone growth.
July 19 (Reuters) - French drugmaker Ipsen (IPN.PA) said on Wednesday the European Commission has not granted marketing authorization for palovarotene, an investigational treatment for fibrodysplasia ossificans progressiva (FOP), a condition that causes abnormal bone growth.
The FDA’s Endocrinologic and Metabolic Drugs Advisory Committee voted in support of the efficacy and risk-benefit profile of Ipsen Biopharmaceuticals’ drug palovarotene, which the agency is reviewing for the treatment of the ultra-rare disease fibrodysplasia ossificans progressiva.
Advisory committee voted 10 for and 4 against that evidence from the Phase III MOVE study show palovarotene is an effective treatment in patients with the ultra-rare bone disease fibrodysplasia...
Post-hoc analyses are generally a major no-no for the FDA, but agency reviewers signaled their openness to considering them in the case of Ipsen’s potential new therapy for patients with an ultra-rare genetic connective tissue disorder.
Palovarotene is the first medicine to be submitted for regulatory approval for fibrodysplasia ossificans progressiva (FOP), an ultra-rare disease with an estimated prevalence of 1.36 per million...
Ipsen (Euronext: IPN; ADR: IPSEY) announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended not to grant marketing authorization for investigational palovarotene as a treatment for the ultra-rare bone disease, fibrodysplasia ossificans progressiva (FOP). In the E.U. there are currently only symptomatic treatments for FOP, which do not reduce the formation of extra-skeletal bone in patients with the condition. Ipsen will be requesting a re-examination of the CHMP opinion, based on scientific data available from the existing palovarotene clinical trial program.
PARIS, FRANCE, 23 December 2022 – The U.S. Food and Drug Administration (FDA) issued a Complete Response Letter (CRL) regarding the New Drug Application for palovarotene, an investigational treatment for the reduction of new abnormal bone formation (heterotopic ossification) in people living with fibrodysplasia ossificans progressiva (FOP). The CRL is related to the regulatory agency’s previous request for additional information on palovarotene clinical trial data communicated to Ipsen in October 2022, which is not a request for additional efficacy or safety data beyond existing studies. Ipsen anticipates responding to the request in the first quarter of 2023 with an expected six-month FDA review cycle. The FDA has not announced a rescheduled date for the Endocrinologic and Metabolic Drugs Advisory Committee meeting for investigational palovarotene.
Less than a week before Ipsen’s palovarotene was due to go in front of an FDA advisory committee, the meeting has been called off following a request by the regulator for more data.
The FDA has scheduled an advisory committee to examine Ipsen’s palovarotene on Oct. 31, according to a notice posted in the Federal Register, setting up a showdown for a drug that once failed a Phase III test. The hearing will come about two months ahead of palovarotene’s decision date, which is slated for Dec. 29.