Pfizer Inc. (PFE - Free Report) announced that the FDA has granted Breakthrough Therapy designation to its rare disease candidate, tafamidis. The candidate is being developed for the treatment of transthyretin cardiomyopathy (TTR-CM).
The designation from the FDA is intended to expedite the development and review of drugs with early evidence of substantial potential clinical benefit to patients, or benefit patients without current treatment options.
The designation for tafamadis was primarily based on encouraging top-line data from the phase III ATTR-ACT study, which demonstrated significant reduction in the combination of all-cause mortality and frequency of cardiovascular-related hospitalizations in TTR-CM patients.
The candidate also enjoys fast track status and orphan drug designation for this indication in the United States.
Shares of Pfizer have declined 0.7% so far this year, comparing favorably with a 4% decrease for the industry.
TTR-CM is a rare disease associated with progressive heart failure and is universally fatal. Presently, no treatment is approved to treat this fatal disease. According to Pfizer’s press release, less than 1% of people with the disease are diagnosed with life expectancy averaging three to five years from diagnosis.
There are two types of TTR-CM, a hereditary form and a wild-type form of the disease, which is not hereditary. The ATTR-ACT study included both types of patients. However, Alnylam Pharmaceuticals (ALNY - Free Report) and Ionis Pharmaceuticals (IONS - Free Report) have developed their candidates, patisiran and inotersen, respectively, for hereditary TTR amyloidosis, or hATTR. Both patisiran and inotersen are under review in the EU and United States and could be launched this year.
Tafamidis is already approved for the treatment of transthyretin familial amyloid polyneuropathy (TTR-FAP) in 40 countries except the United States.
Zacks Rank & Stock to Consider
Pfizer currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
H. Lundbeck (HLUYY - Free Report) is a better-ranked stock in the large cap pharma sector, sporting a Zacks Rank #1.
H. Lundbeck’s earnings estimates increased from $3.20 to $3.57 for 2018 and from $3.24 to $3.38 for 2019 over the last 30 days. The company came up with an average four-quarter positive surprise of 9.8%. The company’s stock has gained 32.4% so far this year.
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