Acorda Therapeutics announced Tuesday that the FDA issued a refusal to file letter regarding the company's marketing application for Inbrija, a self-administered, inhaled powder formulation of levodopa for the treatment of OFF-period symptoms in patients with Parkinson's disease who are on a carbidopa/levodopa regimen. Shares in the drugmaker fell as much as 31 percent on the news.
According to Acorda, the agency identified two reasons for its refusal to review the application. The company said the first matter has to do with the date when the manufacturing site for Inbrija would be ready for inspection, while the second is over "a question regarding the submission of the drug master production record." Acorda noted that the FDA did not request or recommend that it conduct any additional clinical efficacy or safety trials.
CEO Ron Cohen stated "we will work with the FDA as quickly as possible to address the open issues and to clarify the path to successfully re-file our application." He added "we remain confident in Inbrija's data package and its promise as an important new therapy for people with Parkinson's disease."
The company's application for Inbrija, also known as CVT-301, was supported by data from the Phase III SPAN-PD trial. Results from the 339-patient study demonstrated that Inbrija was associated with a significant improvement in motor function versus placebo.
Commenting on the latest news, Leerink analyst Paul Matteis said the FDA's decision is "surprising, puzzling, [and] disconcerting," as the regulator's concerns are not about the drug's data, but rather focus on two "vague" requests about manufacturing. "Even after speaking with the company, the [refusal to file] is mysterious to us," he added. Matteis acknowledged that Inbrija is "intrinsically riskier" given it is a drug-device combination, but "the Phase III data are strong…and the device has undergone over a decade of development, previously as inhaled insulin." Matteis indicated that "the best case scenario would be a resubmission around the end of this year."
Meanwhile, an investor with a 17-percent stake in Acorda recently urged it to consider a sale after a US judge invalidated several patents covering the multiple sclerosis drug Ampyra (dalfampridine), leaving the company with a single patent that expires in the middle of 2018. Following the ruling, which Acorda said it would appeal, the company implemented a restructuring plan that promptly cut its workforce by approximately 20 percent, saying it will focus resources mainly on Inbrija and its Parkinson's disease drug candidate tozadenant.
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