Commercial Alnylam’s RNA drug gets FDA approval as a Pfizer...

Alnylam’s RNA drug gets FDA approval as a Pfizer face-off may be expected


Alnylam’s vutrisiran has gotten the green light from FDA, which may also result in a confrontation with Vyndaqel, Pfizer’s hit franchise in the rare disease market, which is becoming highly competitive.

Vurtisiran, which will be branded in the market as Amvuttra, has been approved by FDA for the treatment of hereditary transthyretin amyloidosis (hATTR) polyneuropathy. This is a disease that is rare and works by weakening and disease harming nerves. U.S. doctors are expected to receive the drug at the start of July, according to the company.

Evaluate Vantage, a platform that provides data-driven insights into Med-tech, biotech, and pharma industry, claims that FDA-approved RNA drug Amvuttra could reach global sales of $1.8B by 2026. This claim is based on the drug’s potential to treat a different type of ATTR, one which affects the heart. This indication is called ATTR cardiomyopathy, and it makes an important field of battle between Pfizer, Alnylam and other players.

Amvuttra is the company’s first FDA approval since the newly hired CEO Yvonne Greenstreet, who joined in January, replacing the founder John Maraganore, Ph.D. Alnylam is well known for RNA interference therapeutics, and Amvuttra belongs to an arising class of the field.

Due to its high metabolic stability, Amvuttra can be used once in three months through under-the-skin injections. For comparison, Onpattro is administered a lot more frequently, which is once every three weeks via intravenous infusion. According to the Chief Commercial Officer of Alnylam, Mr. Tolga Tanguler, Amvuttra is offered at an annual price of $463,500, which is comparable to that of Onpattro.

According to the phase 3 HELIOS-A study, ATTR polyneuropathy patients who took Amvuttra showed an average improvement of 2.2 points after nine months, on a modified neuropathy impairment score. 50% of the patients experienced improvement. In that very study, Onpattro scored 1.4 points improvement.

At the SVB Securities conference in February, Chief Financial Officer of Alnylam, Mr. Jeff Poulton said that due to its convenience advantage, Amvuttra will be the go-to drug for new ATTR patients. However, for the prevailing Onpottra patients, compliance has been high, and they would preferably keep on going with the older medication that they have been taking for years, according to Poulton.

Hereditary ATTR is an uncommon condition that has an impact on 50,000 people globally. On the other hand, non-inherited ATTR, predominantly cardiomyopathy, affects approximately up to 300,000 people worldwide. Some believe this number is under-quoted due to underdiagnoses. Because of this, cardiomyopathy takes commercial precedent over hereditary ATTR.

In 2021, Vyndaqel and Vyndamax, Pfizer’s ATTR therapies, reported $2M in sales, nearly 45.5% of which came from the United States where the only approval it has is for ATTR cardiomyopathy.

Alnylam is running the HELIOS-B study for their drug Amvuttra and the patient survival data is expected a couple of years from now in 2024. In a note to clients dated May, Zhinqiang Shu, Ph.D., an analyst at Berenberg said that by their calculation, Onpattro’s APOLLO-B study does not have a good chance of success. He also shared that more likely to be successful is Amvuttra in its HELIOS-B trial. If successful, it could change current medical practices.

More big players in the drug making market are competing for the ATTR market. AstraZeneca has entered a partnership with Ionis, while Novo Nordisk has acquired a late-stage program from Prothena.

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