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Alnylam receives expected FDA ok for Onpattro

Trust favourite Alnylam approaches key FDA decision

Alnylam receives expected FDA ok for Onpattro

Biotech trust stock favourite Alnylam Pharmaceuticals (Nasdaq: ALNY) received its  first FDA approval late on Friday for Onpattro (patisiran) for the treatment of polyneuropathy in adults with hereditary transthyretin-mediated (hATTR) amyloidosis. However, the FDA did not allow a broader label that included patients with cardiomyopathy (CM), leading to its shares falling by 8% earlier today, even though this was not widely expected by analysts.

Alnylam is held among the sector’s specialist investment trusts including Biotech Growth Trust (BIOG, 3.6% of NAV) and BB Biotech (BBB, 3.6% of NAV). It is also the third largest single holding (3.7% of NAV) for Edinburgh Worldwide Investment Trust (EWI), a diversified non-biotech specialist albeit with significant exposure to healthcare stocks.

The FDA decision’s follows the EMA’s positive opinion last month, which was similarly limited to hATTR PN.  The possibility of including CM stemmed from the fact that Alnylam’s  APOLLO Phase III trial did enrol a subgroup of patients with CM and reported improvements in a variety of cardiac related parameters, but there was no significant improvement in cardiac function as measured by left ventricular ejection fraction.

The approval is nevertheless a landmark event as it is the first for any RNAi based drug. It has also taken Alnylam more than 15 years to develop and required an accumulated R&D spend of more than $2.4bn, although the company has spent money on other product candidates currently in clinical trials. Alnylam intends to market the product itself, setting up its own commercial infrastructure to do so, and plans to charge $450,000 per patient per year in the US – a figure which is not not unusual for a ultra rare disease – which would translate to about $345,000 per patient/year after rebates. hATTR amyloidosis is notoriously underdiagnosed and the company estimating that fewer than 3,000 patients are currently diagnosed in the US. Alnylam estimates some 10-15,000 patients could be candidates for Onpattro once they are appropriately diagnosed.

An important event on the horizon is the presentation of clinical trial data on one of the two competing products in this segment, Pfizer’s Vyndaqel (tafamidis) for transthyretin cardiomyopathy. Earlier this year, Pfizer reported a positive top-line result in its Phase III Attract trial, but reserved full data for presentation at a scientific meeting: this is now expected at the European Society of Cardiology (ESC) Congress on 27 August.

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