- The Food and Drug Administration has issued a refuse-to-file letter regarding the approval application for a blood pressure medicine — the second time in less than a week the agency has used the uncommon rejection method.
- California-based SteadyMed Ltd. announced on Thursday receipt of the letter, which pertained to a New Drug Application (NDA) that the specialty pharma submitted for its pulmonary arterial hypertension (PAH) treatment Trevyent in late June. In the letter, the agency said the NDA was " not sufficiently complete" and called for additional information about testing procedures for the product, among other things.
- "We believe that the issues raised in the letter from FDA can be sufficiently addressed," SteadyMed CEO Johnathan Rigby said in an Aug. 31 statement. "Our next step is to work with the FDA to address the open issues and identify a path to a successful resubmission and acceptance of our application."
Refuse-to-file letters don't come very often. Two in one week may be reflective of the FDA's continued attempts to optimize the drug approval process, in part through rejecting would-be products earlier in the regulatory pathway to save developers, as well as the agency itself, time and resources.
"Incomplete applications can lead to multiple-cycle reviews and inefficient use of [Center for Drug Evaluation and Research (CDER)] resources," the FDA wrote in 2013 regulatory guidelines. "CDER also believes [a refuse-to-file] action can allow an applicant to begin repair of critical deficiencies in the application far sooner than if these were identified much later in a complete response action and may lead to more rapid approval of safe and effective drug products."
Though refuse-to-file letters are less common than complete response letters, their issuance often has similar effects. The most glaring is a sharp decline in the recipient company's stock price; SteadyMed shares, for instance, opened at $3.50 apiece Thursday morning, down more than 40% from the prior day's close.
Other drugmakers that received refuse-to-file letters for their treatments had similar experiences. Earlier this week, a letter addressing Acorda Therapeutics Inc.'s drug for Parkinson’s disease, Inbrija (levodopa), ended up cutting the company's stock value almost in half.
Regarding its letter, SteadyMed said it plans to ask the FDA for a Type A meeting — a meeting centered on righting hold ups in drug development or regulatory approval — within the next 30 days. Such a meeting would allow the company "to gain further clarification on the additional information required for resubmission and acceptance of the NDA," according to the Aug. 31 statement. SteadyMed also said it would give additional guidance about the approval application following that meeting.
Trevyent combines treprostinil, the active ingredient in other PAH products such as United Therapeutics Corp.'s Remodulin and Tyvaso, with SteadyMed's PatchPump, a single-use, two-day patch that is programed to deliver a proper amount of the drug throughout the day. SteadyMed believes its medicine is differentiated from competitor products that can be "multi-component and rather complex."