According to a story from newswiretoday.com, the biopharmaceutical group Ipsen has recently announced its decision to halt its dosing of patients in its phase 3 clinical trial and its phase 2 extension trial. These studies were testing the group’s experimental drug palovarotene as a treatment for fibrodysplasia ossificans progressiva (FOP), a rare disease. The decision was made following the results of a futility analysis, which suggested that the investigational therapy would be unable to reach the phase 3 study’s primary endpoint.
About Fibrodysplasia Ossificans Progressiva (FOP)
Fibrodysplasia ossificans progressiva (FOP) is an extremely rare, severe, and unusual disease. It is characterized by the abnormal transformation of damaged soft tissue into bone. This process is known as ossification. It is the only known medical phenomena in which one organ system is changed into another. It is incredibly rare, with less than 1,000 confirmed cases worldwide. Fibrodysplasia ossificans progressiva is caused by a mutation that affects a gene responsible for tissue repair. This mutation often appears spontaneously and is rarely passed down. Signs and symptoms include deformed big toes at birth, tumor-like lumps, and the slow transformation of soft tissue in bone, which is usually triggered by a flare-up that appears before age ten. The transformation of tissue into bone usually begins from the neck and spreads downward over the years. Patients eventually lose mobility almost entirely, becoming frozen in position. There is no known treatment. Life expectancy is around 40 years. To learn more about fibrodysplasia ossificans progressiva, click here.
The Future of Palovarotene
While the Ipsen’s decision does not bode well for the future of this drug, its ultimate fate still remains to be seen. The US Food and Drug Administration (FDA) previously issued a clinical hold for administration of palovarotene in patients aged 14 years and younger. Despite the review of the futility analysis by the Independent Data Monitoring Committee (IDMC), the organization has urged Ipsen not to discontinue the trial. The IDMC cites disparate data results, concerns about the protocol pre-specified model, and other indicators of possible therapeutic activity as its reasoning for this conclusion.
Ipsen plans to consult with the FDA and continue the assessment of the data gathered so far to decide what will come next for the testing of palovarotene as a treatment for fibrodysplasia ossificans progressiva.