Amyloidosis Drug Competitors Can Coexist While Growing The Market, Alnylam CEO Says
The recent US FDA approval of three drugs – Alnylam’s Onpattro, Ionis’ Tegsedi and Pfizer’s tafamidis – in transthyretin-mediated amyloidosis means more opportunities to increase education and improve diagnosis in this rare disease, Alnylam CEO John Maraganore tells Scrip.
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Health technology appraisal body NICE now says that Alnylam's innovative gene silencing drug is value for money for the treatment of the rare disease, hereditary transthyretin-related amyloidosis.
Tafamidis was approved by FDA in two formulations that will be marketed as Vyndaqel and Vyndamax for the treatment of transthyretin amyloid cardiomyopathy.
Pivotal studies of the first two drugs approved for treating hereditary transthyretin-mediated amyloidosis did not provide cardiac efficacy data on how patients feel, function or survive, reviewers said; the agency approved Alnylam’s patisiran and Ionis’ inotersen for patients with the polyneuropathy form of the disease but not the cardiomyopathy form.