Alnylam Pharmaceuticals ALNY announced that the FDA has accepted its supplemental new drug application (sNDA) seeking the label expansion of Amvuttra (vutrisiran) for review to treat ATTR amyloidosis with cardiomyopathy (ATTR-CM). Amvuttra, an RNAi therapeutic, is ALNY’s lead drug, which is currently approved in the United States for treating adult patients with polyneuropathy of transthyretin-mediated (hATTR) amyloidosis. It is also marketed in the EU for treating hATTR amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy.

The sNDA has been accepted for review under the FDA’s Priority Review pathway. A filing designated as a Priority Review reduces the review period to four months. A final decision from the regulatory body is expected on March 23, 2025. Furthermore, the FDA has informed Alnylam that it does not plan to hold an advisory committee meeting to review the application.

Subject to approval, vutrisiran will become the first therapeutic approved in the United States to treat both the polyneuropathy manifestations of hATTR and cardiomyopathy manifestations of ATTR amyloidosis.

The application seeking approval for Amvuttra to treat ATTR-CM is supported by positive results from Alnylam’s pivotal phase III HELIOS-B study, which met all primary and secondary endpoints across both the overall and monotherapy populations, each with statistical significance.

Year to date, shares of Alnylam have gained 31.1% against the industry’s 9% decline.

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Per the data readout, vutrisiran reduced mortality and cardiovascular events while improving functional capacity (measured by the six-minute walk test), quality of life (using the Kansas City Cardiomyopathy Questionnaire), and heart failure symptoms and severity (via New York Heart Association class) in ATTR-CM patients.

Additionally, the safety profile of vutrisiran in the HELIOS-B study was consistent with its known profile for treating hATTR amyloidosis with polyneuropathy in adults. In the late-stage study, the rates of adverse events, including those leading to discontinuation, were similar between the vutrisiran and placebo groups.

Amvuttra – The Key Growth Driver for Alnylam

Amvuttra sales contribute significantly to Alnylam’s top line. The drug generated sales worth $693.9 million in the nine months ended Sept. 30, 2024, up 79% on a reported basis. The uptake for the product has been encouraging, with new patients starting treatment as well as several patients switching from Onpattro. Notably, Onpattro is approved in the United States and EU to treat hATTR amyloidosis in adults.

We remind the investors that a regulatory filing for vutrisiran, seeking its label expansion to treat ATTR-CM, is also currently under review in the EU. If the regulatory applications in the EU and the United States are approved, Alnylam believes that vutrisiran has the potential to become the new standard of care for the treatment of ATTR-CM. This will expand the eligible patient population for the drug driving substantial growth for the company in the future.

Other marketed products include Givlaari for acute hepatic porphyria and Oxlumo injection for subcutaneous use for the treatment of primary hyperoxaluria type 1 to lower urinary and plasma oxalate levels in pediatric and adult patients.

Alnylam also markets a fifth drug, Leqvio (inclisiran), in collaboration with Novartis NVS to treat hypercholesterolemia in the EU. In the United States, it is approved to reduce low-density lipoprotein cholesterol (LDL-C) with two doses per year. Per the agreement, Alnylam has granted Novartis exclusive and worldwide rights to manufacture and commercialize RNAi therapeutics targeting PCSK9 for treating hypercholesterolemia and other human diseases, including Leqvio. Novartis has also received FDA approval to expand Leqvio’s label to include earlier use in patients with elevated LDL-C who have an increased risk of heart disease, as an adjunct to diet and statin therapy.

Alnylam Pharmaceuticals, Inc. Price and Consensus

Alnylam Pharmaceuticals, Inc. price-consensus-chart | Alnylam Pharmaceuticals, Inc. Quote

FDA Approves BBIO’s Cardiomyopathy Drug

BridgeBio Pharma BBIO recently received FDA approval for its pipeline candidate acoramidis for the treatment of adults with ATTR-CM to reduce cardiovascular death and cardiovascular-related hospitalization. The drug will be launched in the United States under the brand name Attruby. Shares of BBIO gained 16.1% on Monday in response to the news.

The FDA approval is based on positive results from BridgeBio’s phase III ATTRibute-CM study wherein Attruby significantly reduced death and cardiovascular-related hospitalization, and improved quality of life. Per BBIO, Attruby is the first and only approved product with a label specifying near-complete stabilization of TTR. Through near-complete TTR stabilization, Attruby has been shown to preserve the native function of TTR as a transport protein of thyroxine and vitamin A and to demonstrate benefit on cardiovascular outcomes.

The approval boosts BBIO’s portfolio given the potential of the targeted market (market opportunity $15-120 billion). Transthyretin cardiac amyloidosis is a progressive disease with a poor prognosis when left untreated.

A regulatory application is also currently under review in the EU for acoramidis to treat the ATTR-CM indication. A decision is expected in 2025. BridgeBio has granted exclusive rights to Bayer to commercialize acoramidis for ATTR-CM in the EU.

ALNY’s Zacks Rank and Stock to Consider

Alnylam currently carries a Zacks Rank #3 (Hold).

A better-ranked pharma stock is Allogene Therapeutics ALLO, carrying a Zacks Rank #2 (Buy) at present. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

Over the past 60 days, Allogene Therapeutics’ estimates for loss per share have narrowed from $1.40 to $1.35 for 2024, and that for 2025 has narrowed from $1.46 to $1.34. ALLO shares have lost 29% year to date.

Allogene Therapeutics’ earnings beat estimates in three of the trailing four quarters and matched once, delivering an average surprise of 9.42%.

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