Alnylam Gets FDA Nod for Rare Disease Drug

Alnylam Pharmaceuticals (NASDAQ:ALNY) has officially received the nod from the US Food and Drug Administration (FDA) for Givlaari, its treatment for acute hepatic porphyria (AHP) — a rare metabolic disease — the company announced on Wednesday (November 20).

This approval is a significant breakthrough for treating AHP, as there has been no FDA-approved medication for the condition until now. Porphyrias are rare genetic diseases that result in life-threatening neurological attacks including seizures and psychosis.

A press release issued by the federal agency said Givlaari’s approval came following clinical trial results of the company’s ENVISION Phase 3 study, in which 94 patients with AHP participated and received a placebo or the drug.

Wednesday’s approval came less than four months after a new drug application for Givlaari was submitted by the company and accepted by the FDA.

The FDA said that patients dosed with Givlaari had a 70 percent reduction in attacks compared to those who took the placebo. The most common side effects of Givlaari were nausea and localized reactions where the treatment was administered.

“Prior to (Wednesday’s) approval, treatment options have only provided partial relief from the intense unremitting pain that characterizes these attacks,” Richard Pazdur, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research, said in the FDA statement. “The drug approved today can treat this disease by helping to reduce the number of attacks that disrupt the lives of patients.”

Between the US and Europe, there are approximately 3,000 patients diagnosed with AHP. The disease causes a genetic mutation that causes enzymes that assist in hemoglobin function to be unable to be produced. As a result, toxins are built up and lead to stomach and muscle pain that can result in hospitalization. Long-term effects of AHP include chronic neuropathic pain, hypertension, chronic kidney disease and liver disease.

“We believe the approval of Givlaari represents a landmark event for the advancement of precision genetic medicines, providing new hope for patients and their caregivers living with the debilitating manifestations of AHP and unpredictable nature of AHP attacks, as well as for the doctors who diagnose and treat these patients,” John Maraganore, CEO of Alnylam, said in the release.

According to the Boston Business journal, Givlaari will cost patients US$575,000 per year and is required to be taken for life.

In a separate press release also issued on Wednesday, Alnylam detailed a framework for value-based arrangements (VBAs) for patients with AHP. In the release, the company acknowledged that without its treatment, AHP patients can expend between US$400,000 and US$650,000 per year to treat attacks; this includes being hospitalized, hemin administration and other medical procedures.

In the press release, Alnylam said it is conducting conversations with payers regarding VBAs for Givlaari and how to incorporate the drug into plans and negotiations. The company and Harvard Pilgrim have already reached an agreement.

“Under this innovative framework for VBAs, participating government and commercial payers will pay the full value for GIVLAARI only when it delivers patient outcomes in the real-world setting similar to results demonstrated in clinical trials,” the company explained. “An additional and newly designed Prevalence-Based Adjustment (PBA) feature will trigger rebates to participating payers if the number of diagnosed patients they cover exceeds current epidemiologic estimates for AHP.”

Shares of Alnylam Pharmaceuticals rose 10.63 percent throughout the course of Wednesdays’ trading session to close at US$108.21. After-hours trading bumped its price up 0.19 percent to US$108.40 as of 5:09 p.m. EST.

On TipRanks, Alnylam is currently ranked a “strong buy” based off eight analyst ratings, with the most recent rating given on Wednesday. Alan Carr from the firm Needham reiterated his “buy” position with a price target of US$135. Overall, the average price target of Alnylam is US$120.75, with a high of US$150 and a low of US$62.

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Securities Disclosure: I, Jocelyn Aspa, hold no direct investment interest in any company mentioned in this article.