Alnylam's Givlaari Gets Approval In Europe For Rare Disease

Alnylam Pharmaceuticals Inc. (NASDAQ:ALNY) announced that the European Commission has approved Givlaari (givosiran) for the treatment of acute hepatic porphyria (AHP) in patients aged 12 years or older. The drug will be available as subcutaneous injection. The approval in Europe was expected as the drug had received positive recommendation from the Committee for Medicinal Products for Human Use of the European Medicines Agency in January 2020.

The ultra-rare AHP disease can lead to severe abdominal pain, vomiting and seizures, which can be life-threatening due to the possibility of paralysis and respiratory arrest during attacks.

The approval was based on impressive data from the phase III ENVISION study, which showed that treatment with Givlaari resulted in 74% reduction in the rate of porphyria attacks. The drug is the first and only therapy approved in Europe, which demonstrated prevention of AHP attacks, reduction in chronic pain and improving quality of life.

Please note that Givlaari was approved by the FDA in November last year for the same indication. It has already been launched in the United States and generated $0.2 million in the fourth quarter of 2019. The drug is priced at $575,000 per annum in the U.S. market.

Shares of the company have rallied 31.1% year to date against the industry’s decrease of 7.7%.

Alnylam is focused on development of novel RNAi-based therapeutics targeting primarily rare-disease indications. The company has several collaborations with leading pharmaceutical and life sciences companies including Novartis (NYSE:NVS) , Roche (OTC:RHHBY) and Ionis Pharmaceuticals (NASDAQ:IONS) among others. It has received approval for two RNAi-based therapeutics — Givlaari and Onpattro — in the United States and Europe in the last 18 months.

Onpattro was approved in 2018 for the treatment of polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. The drug generated $166 million in 2019. Apart from its approved drugs, Alnylam is developing several RNAi-based candidates. The company has initiated new drug application (“NDA”) rolling submission for lumasiran, with remaining sections expected to be submitted to the FDA in early 2020. The NDA is seeking approval for lumasiran as a treatment for primary hyperoxaluria type I, a rare-disorder affecting kidneys.

Further, the company expects to advance inclisiran, being evaluated to treat hypercholesterolemia, along with partner Novartis and filed an NDA in December 2019. It is also developing another candidate, fitusiran, in collaboration with Sanofi (PA:SASY) in late-stage study as a treatment for hemophilia A or B.

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Alnylam currently has 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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