NHS England and Biogen Sign Agreement on Costly Medication for Fatal Disease

NEW YORK (Reuters) – Britain's National Health Service has announced an agreement to pay for expensive Spinraza treatment by US drug maker Biogen Inc.

NHS England has announced a Controlled Access Agreement with Biogen, which will fund a treatment for a limited time and collect data on the effectiveness of the drug.

NHS England has not revealed the price to pay for the drug.

Biogen confirmed this deal Tuesday and welcomed the decision of the National Institute for Health and Wellness Excellence to recommend funding for Spinraza in the UK.

Spinraza, given every four months by injection into the spine, has a US list price of $ 750,000 (£ 581,122) for the first year and $ 375,000 per year thereafter.

The UK health care agency, NICE, said in August that it could not recommend Spinraza as a cost-effective treatment. The rejection came despite a British price tag of less than £ 450,000 in the first year and Biogen's offer of an undisclosed rebate to the National Health Service.

Spinal muscular atrophy (SMA) is the leading genetic cause of infant mortality, affecting one in 10,000 births. About 60% of patients have the most severe form of type 1, which often leads to paralysis, respiratory failure and to death at the age of two years.

According to the NHS England, between 600 and 1,200 children and adults currently live with this disease in England and Wales. Spinraza will be immediately made available to type 1 patients by Biogen, he said.

Spinraza will be made available to other patients – including adults and siblings who have not yet presented symptoms – in a few weeks, after the publication of NICE guidelines.

Spinraza was the first approved treatment for ADM, but will soon face new competition. The US Food and Drug Administration is set to approve Zolgensma's Novartis gene therapy this month, and other regulators are expected to do so later this year.

Novartis argues that spot treatment could be a cure for ADS and priced at between $ 1.5 million and $ 5 million. This would make Zolgensma the most expensive new therapy.