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Roche scores FDA priority review for risdiplam in SMA

Potential rival to Novartis’ Zolgensma and Biogen’s Spinraza


Roche has been granted a priority review from the FDA in the US for its spinal muscular atrophy therapy risdiplam, setting up a potential market clash with rivals from Novartis and Biogen. 

Roche recently revealed positive phase 3 data showing risdiplam can improve motor function in patients with SMA – a muscle-wasting disease that affects one in 6,000 to 10,000 live births and is a leading cause of genetic deaths in infants.

In severe cases, patients become paralysed and lose the ability to carry out basic functions such as swallowing and breathing.

The results of Roche’s SUNFISH study demonstrated that following a year’s treatment with its drug, patients aged two to 25 with SMA type 2 or type 3 had better Motor Function Measure 32 (MFM-32) scale scores compared to placebo.

The priority review means that the filing for risdiplam will be fast-tracked, with a final decision on approval expected by 24 May 2020.

In addition to the SUNFISH study of type 2 and type 3 SMA patients, Roche is currently investigating risdiplam in the FIREFISH clinical trial in infants with type 1 SMA.

“The FIREFISH and SUNFISH trials were designed to represent the real world spectrum of people living with SMA and include many people previously underrepresented in clinical trials,” said Levi Garraway, chief medical officer and head of global product development at Roche.

“We look forward to working closely with the FDA to explore broad access to risdiplam for all individuals in the community who might benefit,” he added.

If Roche scores approval for the drug, it will compete with Biogen’s blockbuster antisense-based SMA medicine Spinraza (nusinersen) and Novartis’ one-shot gene therapy Zolgensma (onasemnogene abeparvovec).

These drugs both come with high price tags – Spinraza costs $750,000 in the first year of treatment and $375,000 thereafter in the US, and Zolgensma costs $2.1m, the most expensive drug in the world.

Roche’s risdiplam is a closer competitor to Biogen’s drug, based on its target age range. It could however have a dosing advantage – risdiplam is an orally-active treatment, while Spinraza is injected into the spine.

Although, Roche’s drug has to be taken every day while Biogen’s treatment is dosed every four months after initial injections.

Article by
Lucy Parsons

25th November 2019

From: Regulatory



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