Roche drug RG7916 granted Orphan Drug Status in the US

17 January 2017

The US Food and Drug Administration (FDA) has granted Orphan Drug Status to RG7916, a splice-modifying drug developed through a partnership between Roche, PTC Therapeutics, and the SMA Foundation.

Orphan Drug Status gives incentives to companies to pursue drugs intended to treat rare conditions with small markets, such as SMA, and can help to expedite the lengthy process of drug development and approval.

RG7916 is a small molecule that targets and encourages the SMN2 backup gene to produce a greater amount of functional SMN protein, which is lacking in people with SMA. By increasing SMN levels, RG7916 may be able to improve some of the symptoms caused by the disease.

RG7916 can be taken by mouth, meaning that should it ultimately be approved for use in SMA patients, it would be much easier to administer on a large scale.

Results should soon be posted from a 2016 Phase 1 trial of RG7916, designed to assess its safety in healthy volunteers (click here for more information).

Roche recently announced that they are actively recruiting SMA patients across Europe for two Phase 2 clinical trials of RG7916 (click here for more information). These trials, named FIREFISH and SUNFISH, will be conducted in patients with SMA Type 1 and Types 2/3, respectively.  A third trial called JEWELFISH is scheduled to begin in early 2017 and will assess safety, tolerability, and pharmacokinetics of RG7916 in people aged 12-60 years with Type 2 or 3 SMA.

Further Information

RG7916 press release

FIREFISH trial page

SUNFISH trial Page 

Phase I trial page