Alnylam, PTC Therapeutics rare disease drugs win backing from England's NICE

Despite high list prices, England's National Institute for Health and Care Excellence (NICE) has recommended rare disease treatments from PTC Therapeutics and Alnylam Pharmaceuticals. 

With the endorsements, PTC’s Translarna and Alnylam’s Amvuttra will soon be available for patients through England's National Health Service (NHS). The drugs treat nonsense mutation Duchenne muscular dystrophy and hereditary transthyretin-related amyloidosis (hATTR), respectively.

Over the last decade, PTC's Translarna has traveled a long and winding road. Back in 2014, Europe's Committee for Medicinal Products for Human Use (CHMP) rejected the drug after it missed the mark in a phase 2b trial. PTC appealed that decision and eventually scored a European approval for its medicine.

Further, the drug has been rejected three times by the FDA, with the latest coming in 2017. The company hasn’t given up on an FDA approval, touting data in June that CEO Stuart Peltz said could support another application.

For the last six years, the therapy has been available under a managed access agreement in England, meaning patients could have access to the drug while officials collected more data, NICE said in a statement. Through the agreement, which officially ends Friday, around 60 children received the drug. Between 60 and 70 children are born every year in England with the disease, with it being caused by a "nonsense mutation" in around 13%, or six to nine of them.

Now, data show that the treatment is “likely” to slow down disease progression, including delaying the point at which patients lose the ability to walk, NICE said. Still, while evidence for later stage disease is “limited and highly uncertain,” NICE experts agreed that the drug might improve outcomes after patients lose their ability to walk. The drug costs 220,000 pounds sterling per patient per year before any discounts.

Amvuttra, meanwhile, treats a patient population of about 150 people in the United Kingdom. It’s recommended as an option for patients with mild symptoms of hATTR who can walk or those who have moderate symptoms and require assistance to walk. It costs 95,862.36 pounds per injection, with an estimated annual cost per patient of 383,449.44 pounds before any discounts. Alnylam inked a “confidential commercial” discount to make the treatment available to the NHS, NICE said in a recent note.

That drug one of the first recommended under NICE’s new "proportionate approach to technology appraisals," which the agency created to allow a more flexible approach to appraisals. In this case, the simplified process resulted in a final draft guidance produced 20 weeks faster (nearly 60%) than the standard process, NICE said.

In the new approach, the treatment's cost and benefits were compared against Onpattro, another Alnylam drug that treats polyneuropathy in hATTR patients. NICE found that Amvuttra works just as well as Onpattro and costs significantly less per patient thanks to an infusion schedule of every three months compared with every three weeks.

Final guidance for Amvuttra will be published in February, barring any appeals, NICE said.

The NICE endorsements come after the agency recently rejected five COVID-19 products due to concerns over either their costs or effectiveness.