Biopharmaceutical stock Alnylam Pharmaceuticals (NASDAQ:ALNY) is riding a tsunami of hope that a treatment for a rare heart condition receives Food & Drug Administration approval. Although it has several drugs already on the market, the biopharma is not yet profitable.

But if Alnylam can gain expedited approval from the FDA for its RNA interference drug vutrisiran, that could change quickly. Alnylam stock soared 31% on positive Phase 3 trial news and continued rising afterward. Getting vutrisiran, which is marketed as Amvuttra, to market for this new condition just might make profitability a reality.

The question for investors is whether Alnylam stock has wrung all the gains out of the positive developments. Is ALNY a “buy the rumor, sell the news” type of stock?

Ready to Take on the Giants

In late June, Alnylam Pharmaceuticals reported success in treating a progressive and deadly form of heart disease. Amvuttra showed “a statistically significant reduction in the composite of all-cause mortality and recurrent cardiovascular events” in patients with transthyretin amyloidosis with cardiomyopathy, or ATTR-CM. 

The goal was for vutrisiran to reduce death and hospitalizations in patients who were taking Pfizer’s (NYSE:PFE) tafamidis that is approved for treating the condition. It was also tested for those not on the drug.

Alnylam said there was significant improvements in secondary measures of heart health in both populations. Vutrisiran achieved 28% and 33% reduction in “composite of all-cause mortality and recurrent cardiovascular events in the overall and monotherapy populations, respectively.”

It achieved even better results in secondary endpoints of the trials with 36% and 35% reductions, respectively.

“We really feel that this data has set the bar for a new standard of care,” Alnylam CEO Yvonne Greenstreet said in an interview with Bloomberg.

The strong results is encouraging Alnylam to ask the FDA for an expedited review of the results by the end of the year. Amvuttra could hit the market by early 2025.

This is a major achievement for Alnylam Pharmaceuticals. If the FDA agrees, the biopharma could have a major blockbuster drug on its hands over the next decade.

A New Way to Combat Disease

Alnylam is a major player in designing RNA interference therapies. RNAi is a naturally occurring process in the body. It stops genes from overexpressing, which can lead to protein misfolding, or a condition where proteins are unable to naturally fold due to mutations in the amino acid sequence or other external factors.

The biopharma received the first-ever approval of an RNAi therapeutic in 2018. Patisiran, which is marketed as Onpattro, was approved for polyneuropathy in hereditary ATTR amyloidosis.

The following year, it was approved to market Givlaari for acute hepatic porphyria. In 2021, Leqvio, which was developed in partnership with Novartis (NYSE:NVS) for the treatment of high cholesterol, gained approval.

Alnylam’s received approval for its fourth drug, Amvuttra for the treatment of ATTR amyloidosis with polyneuropathy, in 2022.

Last year, Alnylam generated $1.24 billion in net product revenue from these therapies, or 39% growth over the prior year. Yet it still reported a $440 million GAAP loss, or $201 million on an adjusted basis.

Vutrisiran could change all that.

Blockbuster Status on the Horizon

Using the molecular technology, Alnylam could steal a large share of the market away from Pfizer. The drugmaker generated over $3.3 billion in sales of tafamidis, which it markets as Vyndaqel, Vyndamax and Vynmac.

Moreover, because Alnylam tested the drug for patients not on tafamidis, that could vastly expand the universe of patients for the biopharma.

The one word of caution for investors is that Alnylam didn’t release the full results of its study. That leaves some opaqueness to the full achievement.

Since the biopharma is moving forward with a request for an expedited review, it suggests there are no skeletons skulking in the shadows but it is something to be mindful of.

A lot depends on the commercial launch and patient adoption of Amvuttra. Yet this preliminary look at Alnylam stock suggests this is a biopharmaceutical well worth investing in for the long haul.

On the date of publication, Rich Duprey did not hold (either directly or indirectly) any positions in the securities mentioned in this article. The opinions expressed in this article are those of the writer, subject to the InvestorPlace.com Publishing Guidelines.

On the date of publication, the responsible editor did not have (either directly or indirectly) and positions in the securities mentioned in this article.