With Pfizer's tafamidis data, there's a new kid on the ATTR block

Print 30 March 2018
Amirah Al Idrus / FierceBiotech

Pfizer reported positive results from a phase 3 trial of tafamidis in transthyretin cardiomyopathy, a form of the rare disease transthyretin amyloidosis (ATTR). The news drove down stocks for Alnylam and Ionis, both of which are developing drugs for hereditary forms of ATTR: patisiran and inotersen. 

ATTR is caused by a mutation in the TTR gene, resulting in abnormal TTR proteins that fold incorrectly. Symptoms usually include neuropathy as well as cardiomyopathy. There is no approved treatment yet for TTR cardiomyopathy.

“These topline results are important for people with transthyretin cardiomyopathy and bring us one step closer to realizing the potential for a new treatment for those in desperate need,” said Brenda Cooperstone, M.D., senior vice president and chief development officer of rare disease at Pfizer Global Product Development. "We look forward to sharing the detailed results of the study with the cardiovascular community and discussing these data with health authorities to determine an appropriate regulatory path forward.” 

It hasn't been all smooth sailing for Pfizer, which acquired tafamidis in 2010. It filed for approval based on data from a single study in TTR familial amyloid polyneuropathy (TTR-FAP), which resulted in a rejection from the FDA in 2012. The drug is marketed as Vyndaquel in the European Union, where it is approved to delay peripheral neurologic impairment in patients with stage 1 TTR-FAP. 

Just yesterday, Alnylam unveiled late-phase data showing patisiran improved cardiomyopathy. And last September, a phase 3 readout showed that the treatment beat out placebo in improving neuropathy impairment. Alnylam and Ionis both presented phase 3 data in November, with patisiran's 34-point improvement against placebo edging out inotersen's 20-point difference. 

Having rejigged its deal with Sanofi to to take full control of patisiran, Alnylam expects to commercialize the treatment for hereditary ATTR this year, while Ionis recently teamed up with its spinoff, Akcea, to bring inotersen to market. The collaboration came seven months after its previous partner, GlaxoSmithKline ditched Ionis as the pair were closing in on a regulatory filing for inotersen.

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