Stock Alert: Alnylam Pharma's Porphyria Treatment Receives European Ok

(RTTNews) - RNAi therapeutics company Alnylam Pharmaceuticals, Inc. (ALNY) said it received European approval for its drug GIVLAARI to treat acute hepatic porphyria or AHP, an ultra-rare genetic condition that causes debilitating attacks of severe abdominal pain, vomiting and seizures. The marketing authorization in Europe allows GIVLAARI to treat AHP in adults and adolescents aged 12 years and older.

The European approval is based on a phase 3 study, in which the drug demonstrated a 74% reduction in the annualized composite rate of porphyria attacks in AIP patients vs. placebo. John Maraganore, CEO of Alnylam, stated, that there are no currently approved medicines in Europe to decrease the frequency of attacks. GIVLAARI, which received FDA endorsement in November, last year, received strong initial interest subsequent to approval.

Drugs based on Alnylam's RNAi therapeutic platform work on the 'gene silencing' method, by potently silencing the genetic precursors that encode for disease-causing proteins, thus preventing them from being made.

The company's other drug on the market Onpattro treats polyneuropathy of hATTR amyloidosis in adults, a rare, progressive, fatal neurological disease. Onpattro fetched Q4 and FY19 revenues of $55.8 Million and $166.4 Million, respectively.

ALNY is definitely a stock to watch, as the company prepares for potential approval of a third possible product - Lumasiran to treat primary hyperoxaluria, another ultra-rare disease that causes Calcium buildup in kidneys and recurrent kidney stones.