With generics threatening its main moneymaking medication, and an investor threatening its independence, Acorda Therapeutics has been pinning its hopes on approval of a new Parkinson’s disease drug. But that is now in jeopardy because of questions the FDA has about its manufacturing readiness.
The FDA delivered Acorda a Refusal to File (RTF) letter for Inbrija, a self-administered, inhaled formulation of levodopa for treating Parkinson’s disease, the Ardsley, New York-based company reported today, leading its shares to fall more than 25%.
The company said the FDA has two issues with its NDA, saying there were questions about when its manufacturing site would be ready for a preapproval inspection and over its drug master production records. Acorda said the FDA wanted some other info as well, but the FDA indicated that was not specific to the RTF.
The drugmaker said it believes the issues are “addressable" and is seeking a Type A meeting with the FDA as soon as possible to clarify the agency’s concerns. Acorda pointed out that the agency had not recommended additional clinical efficacy or safety studies.
Leerink Research analyst Paul Matteis told clients in a note today that he found the situation “a very significant surprise” for the drug candidate, which Leerink had handicapped as having a 90% chance for approval. While it is a drug-device combo, so “intrinsically riskier,” Matteis said the phase III data are strong, and the device has undergone more than 10 years of development, previously for inhaled insulin.
He said he had spoken with the company execs which told him they believe Acorda is ready for a pre-approval inspection but were unable to shed much light on the FDA’s concern over master production records.
The drug is slated to be commercially produced at a 90,000-square-foot manufacturing facility in Chelsea, Massachusetts, where clinical supplies of CVT-301 were produced by Civitas Therapeutics, which Acorda bought in 2014 to get hold of the drug. The inhalers for it are produced by a contractor.
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The RTF comes at a difficult juncture for the drugmaker, which laid off 20% of its workforce, or more than 100 staffers, in April as it hunkers down in the face of generic competition for its main money-maker, $500 million-seller multiple sclerosis med Ampyra.
Commercial and manufacturing were largely spared in the cuts, which hit R&D hard as the drugmaker reduces the emphasis on earlier-stage programs so it could focus on its Parkinson’s disease drug candidates tozadenant and, of course, Inbrija.
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But one of Acorda’s investors thinks it should take a different tack. The hedge fund Scopia Capital Management earlier this month in a letter to the board began pushing for Acorda to put itself on the block, arguing that its value is up now but will be hurt by generics.