NICE asks for further evidence for PCT's DMD drug Translarna

In draft guidance produced by its Highly Specialised Technologies program, England and Wales medicines watchdog the National Institute for Health and Care Excellence (NICE) has today asked for further clarification from US biotech firm PCT Therapeutics (Nasdaq: PTCT) on the size of the benefit its drug Translarna (ataluren) provides in the treatment of a type of Duchenne muscular dystrophy (DMD).

The NICE has asked the company to provide the results of a confirmatory study of ataluren as soon as possible. The draft guidance also asks the company to provide further justification for the cost of the drug. The draft guidance relates to people with DMD with a mutation (known as a nonsense mutation) in the dystrophin gene. Around eight–13 boys are born with the condition each year in the UK.

Current treatment options for patients with DMD in England are limited and aim to alleviate symptoms and manage complications. These include cardiac and respiratory monitoring and support, orthopaedic intervention and spinal surgery. The current mainstay of treatment, corticosteroid therapy, can cause serious unwanted effects such as growth retardation, bone thinning, mood swings and weight gain.

Ataluren is the first licensed treatment for DMD that addresses the loss of dystrophin, the underlying cause of the condition. It has a conditional marketing authorization in the UK for the treatment of DMD resulting from a nonsense mutation in the dystrophin gene, in patients who are able to walk aged five years and older.

Drug represents an important development, but cost still a question