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Pfizer Gets FDA's Priority Review for Rare Disease Candidate

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Pfizer, Inc. (PFE - Free Report) announced that the FDA has granted priority review to a new drug application (NDA) looking for approval of tafamidis. The pipeline candidate has been developed to treat transthyretin amyloid cardiomyopathy (ATTR-CM), a rare and fatal illness associated with progressive heart failure.

Pfizer filed two NDAs for tafamidis, one for the meglumine form (20 mg capsule) and the other for a free acid form (61 mg capsule). The priority review designation is for the meglumine form with the FDA’s decision on this NDA expected in July this year. The FDA decision for the free acid form is expected in November with the filing being given a standard review period.

There are two types of TTR-CM, a hereditary form and a wild-type form of the disease, which is not hereditary. 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.

The NDA filings are based on data from a phase III study, ATTR-ACT, which evaluated oral daily dose of 20 mg or 80 mg tafamidis meglumine versus placebo in patients with wild-type or hereditary ATTR-CM. The ATTR-ACT study included both types of patients.

Data from the study has shown that treatment with tafamidis led to a significant reduction in the combination of all-cause mortality and frequency of cardiovascular-related hospitalizations — the primary endpoint — compared with placebo at 30 months. Treatment with tafamidis led to a 30% reduction in the risk of mortality and 32% reduction in the rate of cardiovascular-related hospitalization compared to placebo. Tafamidis also helped in reducing the rate of decline in functional capacity, as measured by a six-minute walk test distance and also the rate of decline in aspects of quality of life compared to placebo.

Shares of Pfizer have rallied 15.7% in the past year compared with the industry’s 0.6% increase.



Tafamidis enjoys Orphan Drug, Fast Track and Breakthrough Therapy designations in the United States for ATTR-CM

Please note that tafamidis, on approval, will face competition from Alnylam Pharmaceuticals’ (ALNY - Free Report) Onpattro (patisiran) and Akcea Therapeutics, Inc. (AKCA - Free Report) /Ionis Pharmaceuticals, Inc.’s (IONS - Free Report) Tegsedi (inotersen). Both the drugs were approved in the United States in mid-2018 for treating hereditary TTR amyloidosis or hATTR. However, while tafamidis has been developed to treat both the hereditary and the wild-type form of TTR-CM, Onpattro and Tegsedi have been approved to treat only the hereditary form of the rare disease.

After Pfizer revealed that its new drug application for tafamidis has been granted priority review, shares of Alnylam, Ionis and Akcea declined more than 5%, 2% and 3%, respectively, on Monday.

Tafamidis is already marketed by the brand name of Vyndaqel for the treatment of transthyretin familial amyloid polyneuropathy (TTR-FAP) in 40 countries except United States. In the United States, Pfizer received a complete response letter (CRL) from the FDA on for TTR-FAP in 2012.

Pfizer currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

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