July 25, 2024

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Pfizer encountered the strongest opponent in this field of rare diseases

Pfizer encountered the strongest opponent in this field of rare diseases


Pfizer encountered the strongest opponent in this field of rare diseases.

Recently, Alnylam Pharmaceuticals’ RNAi therapy Amvuttra (vutrisiran) received FDA approval for the treatment of polyneuropathy with hereditary transthyretin-mediated amyloidosis (hATTR) in adults .


The FDA approval was based on positive 9 -month results from the Phase 3 HELIOS-A study , in which Amvuttra significantly improved signs and symptoms of polyneuropathy, with cessation or reversal of disease manifestations in more than 50% of patients.



Pfizer encountered the strongest opponent in this field of rare diseases


The approval of Amvuttra as a follow-up therapy to Alnylam ‘s similar product Onpattro (Patisiran) will join forces with Pfizer’s blockbuster Vyndaqel (meglumine meglumine) in the increasingly competitive hATTR rare disease field.


Onpattro is the first RNAi therapy approved by the US FDA and EU in 2018 for the treatment of hATTR .

While Onpattro is administered by intravenous infusion every three weeks, Amvuttra can be administered less frequently by subcutaneous injection every three months due to its high metabolic stability. Alnylam will offer the Amvuttra at an annual list price of $ 463,500 , in line with the Onpattro ‘s list price.


Vyndaqel was approved by the FDA in May 2019 for the treatment of hereditary or artificial transthyroid hormone-mediated cardiomyopathy, mainly caused by amyloidosis.


Based on Amvuttra ‘s potential to treat another type of cardiac ATTR , Evaluate Vantage recently predicted that global sales of the drug could reach $ 1.8 billion by 2026 .


The key market players for this indication, called ATTR cardiomyopathy, are Alnylam and Pfizer.


Alnylam CFO Jeff Poulton said in February that new ATTR patients may start using Amvuttra because of its convenience benefits .

But for existing Onpattro patients, adherence has been high, and they may prefer to stick with their years-old medication.


Inherited ATTR , including polyneuropathy and a small subset of cardiomyopathy, is a rare disorder that affects approximately 50,000 people worldwide.

But non-hereditary ATTR , primarily cardiomyopathy, affects an estimated 200,000 to 300,000 people worldwide.


Last year, Pfizer’s ATTR therapies Vyndaqel and Vyndamax had combined sales of $ 2 billion , of which $ 909 million came from the U.S.

The drugs are only approved for ATTR cardiomyopathy, which are labeled polyneuropathy in the rest of the world.


Despite being a year ahead of Vyndaqel in the U.S. market , Onpattro ‘s total sales in 2021 will only be $ 475 million based on its polyneuropathy indication .


As for use in ATTR cardiomyopathy, Alnylam is conducting the HELIOS-B study for Amvuttra and expects patient survival data in 2024 .

For now, investors are anxiously awaiting preliminary Phase 3 APOLLO-B data from Onpattro in this heart disease later this year.


Industry watchers are primarily concerned that APOLLO-B will fail on its primary endpoint, which measures the change in 6 -minute walk distance after 12 months of treatment.


Notably, in December last year , BridgeBio ‘s Phase 3 ATtribute-CM study of acoramidis in transthyretin amyloid cardiomyopathy (ATTR-CM) failed to meet its primary endpoint at 12 months.


Pfizer encountered the strongest opponent in this field of rare diseases


Other big pharma companies are also aggressively vying for ATTR market share.


In December , AstraZeneca received a $ 200 million upfront payment, up to $485 million in development and approval milestones, plus another $ 2.9 billion payment tied to sales, to secure Ionis Pharma’s bid for Eplontersen (formerly known as Ionis- trr-lrx ).


Eplontersen is a ligand-binding antisense investigational drug currently in Phase 3 clinical trials in amyloidosis thyroxin cardiomyopathy or ATTR-CM and amyloid thyroxine polyneuropathy or ATTR-PN .


In July last year , Novo Nordisk acquired Prothena ‘s anti-amyloid immune antibody drug PRX004 in clinical development and its transthyretin amyloidosis ( ATTR ) program.


Under the terms of the agreement, Prothena will receive up to $ 1.2 billion in R&D and sales milestone payments, including up to $ 100 million in upfront payments and near-term clinical milestone payments.






Pfizer encountered the strongest opponent in this field of rare diseases

(source:internet, reference only)

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