EMA Regulatory Committee Grants Accelerated Assessment to Patisiran to Treat FAP

EMA Regulatory Committee Grants Accelerated Assessment to Patisiran to Treat FAP

Alnylam Pharmaceuticals’ lead investigational therapeutic patisiran, for the treatment of hereditary ATTR (hATTR) amyloidosis, also known as familial amyloid polyneuropathy (FAP), was granted accelerated assessment by the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP).

“We are pleased the CHMP has granted accelerated assessment for patisiran and believe this underscores the urgent need to improve outcomes for patients living with hATTR amyloidosis,” Eric Green, vice president and general manager of the TTR program at Alnylam, said in a press release.

Alnylam is planning to request commercialization authorization for patisiran in the European Union (EU) by the end of 2017. This new status will expedite the mandatory review process of the marketing application from 210 to 150 days.

The accelerated assessment status is granted to therapies considered to be therapeutic innovations that will cover a public health need in order to provide access to therapies more quickly.

“The news that patisiran has been accepted for accelerated assessment by the EMA signals to us that everyone involved recognizes the serious impact hereditary ATTR amyloidosis has on the lives of people affected by this devastating, progressive disease,” said Eric Low, chairman of the Amyloidosis Research Consortium UK. “The need for new treatment options is urgent. The patient community really welcomes when everything possible is being done to assess promising new treatments quickly and efficiently to see if patients may benefit earlier.”

Patisiran is an investigational therapy developed by Alnylam in collaboration with Sanofi Genzyme. It was designed to bind to mRNA molecules that encode the transthyretin (TTR) protein, therefore blocking its production. This process impairs the production of TTR amyloid clusters in the peripheral nerves, potentially restoring their function and preventing the progression of FAP symptoms.

The investigative therapy was evaluated in the APOLLO Phase 3 study (NCT01960348) that included 225 patients with FAP. Of those, 148 received patisiran and 77 received a placebo.

Treatment with the investigative drug for up to 18 months was shown to improve the modified neuropathy impairment score (mNIS+7) by 34 points compared to a placebo. In addition, 56% of patisiran-treated patients experienced a reduction of impairment scores relative to baseline, compared to 4% of patients that received a placebo. These results suggest that patisiran can effectively improve FAP patients’ physical functions.

The findings were followed by a significant improvement in the patients’ quality of live, as shown by an improvement of 21.1 points in the Norfolk Quality of Life-Diabetic Neuropathy score (Norfolk-QOL-DN) in the treated group compared to those in the placebo group. Also in this assessment, 51% of patisiran-treated patients showed improved Norfolk-QOL-DN scores relative to baseline.

The investigative treatment also demonstrated encouraging effects on cardiac function in patients affected by amyloidosis in the heart.

Patisiran was found to be overall safe and well-tolerated, with the most common adverse effects reported being peripheral swelling and infusion-related reactions.

“The results from the APOLLO Phase 3 study in hATTR amyloidosis patients with polyneuropathy provide evidence that patisiran has the potential to improve neurologic impairment, quality of life, and cardiac parameters,” Green said. “With this accelerated assessment award from the EMA, we hope to be able to make this promising treatment option available to patients in Europe sooner.”

Alnylam plans to submit a marketing applicationfor patisiran with the U.S. Food and Drug Administration (FDA) by the end of 2017. Sanofi Genzyme is also preparing for regulatory approval applications for this new therapy with entities in Japan, Brazil, and other countries during the first half of 2018.

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