Merck (MRK) revealed on Wednesday that the U.S. FDA has rescinded a Breakthrough Therapy Designation for its investigational Hepatitis C treatment.
This is a rare move by the FDA (the first of its kind, actually), but is in line with the designation’s intent: to expedite the approval process for novel medicines that meet significant unmet needs. In the notification to Merck, the FDA cited the availability of other recently approved treatments for HCV. Essentially, the FDA is following through on its mandate to apply the designation only to “true“ breakthroughs.
Merck also reported disappointing 4Q sales and guided for 2015 earnings slightly below analyst expectations. Merck said it expects 2015 EPS of $3.32 – $3.47; Wall Street had expected $3.49 per share. As with many multinationals reporting in the last two weeks, the company cited the strong dollar’s negative impact in 2015.
Merck still plans to file a New Drug Application for grazoprevir/elbasvir (MK-5172/MK-8742) in the first half of this year but plans to discuss the rescission – the first of its kind – with the FDA. Merck does not expect this will impact its ability to, or the timing of, a filing. The product will join other all-oral cures for HVC including Gilead Sciences’ (GILD) Sovaldi/Harvoni and AbbVie’s (ABBV) Viekira Pak.
Though the first rescission to date, the process was built into the legislation to allow for removal when the circumstances permit. From the FDA’s Guidance for Industry on expedited programs:
When breakthrough therapy designation is no longer supported by emerging data or the designated drug development program is no longer being pursued, the Agency may choose to send a letter notifying the sponsor that the program is no longer designated as a breakthrough therapy development program. Consistent with FDA’s commitment to communicate frequently, and in an interactive manner, with sponsors of drugs designated as breakthrough therapies, FDA will notify the sponsor of its intent to rescind and will offer the sponsor an opportunity to justify its product’s continued designation. FDA recognizes that sponsors of products that have had their breakthrough therapy designation rescinded because available data no longer support the designation may still have sufficient evidence after completion of the drug development program to support marketing approval.
Frankly, it’s good to see the FDA taking the designation, or lack thereof, seriously.
