Inotersen a Step Closer to Approval as FDA Grants Priority Review to Ionis’ NDA

The U.S. Food and Drug Administration (FDA) has agreed to speedily review Ionis Pharmaceuticals’ application to get  its investigational drug inotersen approved as a treatment for hereditary TTR amyloidosis (hATTR).

The application has been granted Priority Review status, shortening the review process from 10 to six months. The FDA is expected to make a decision on the application by July 6, 2018.

“TTR amyloidosis is a progressive, fatal disease with very limited treatment options,”  Sarah Boyce, chief business officer of Ionis Pharmaceuticals, said in a press release.

“Receiving Priority Review in the U.S. and Accelerated Assessment in the EU for inotersen shows that the regulatory agencies recognize the high unmet need and the urgency to identify effective therapies to treat patients with this devastating disease,” she added.

Inotersen is a so-called antisense drug — it silences the production of faulty transthyretin, or TTR. Accumulation of TTR in the tissues causes hATTR, also called familial amyloid polyneuropathy (FAP).

Several months ago, Ionis submitted an application to the European Medicines Agency (EMA), which has also granted the treatment accelerated assessment.

“We have been working closely with both the FDA and the EMA following the completion of our Phase 3 study to expedite the regulatory review timelines for inotersen,” said Brett Monia, PhD, senior vice president of drug discovery and franchise leader for oncology and rare diseases at Ionis Pharmaceuticals.

The Phase 3 NEURO-TTR study (NCT01737398), completed in May 2017, met both its primary objectives by improving patients’ quality of life and reducing the severity of neuropathy. Benefits were seen in patients with different mutations and varying disease severity.

While the safety of the treatment was considered good, two main issues were identified. Patients treated with inotersen were at risk of reduced platelet counts and kidney problems. The study found that close monitoring and supportive therapies could stymie the impact of these side effects.

“Today’s outcome … reflects the potential for inotersen to serve a major unmet medical need based on the results of our Phase 3 study, which demonstrated early and sustained benefit to patients treated with inotersen in multiple aspects of their disease, including quality of life,” Monia said.

“We continue our launch preparations for inotersen and are on track to launch inotersen promptly in the U.S. and E.U. if approved,” Boyce added.