Rare Disease Report

Alnylam's Givosiran for Porphyria Patients Gets FDA Breakthrough Therapy Designation

MAY 30, 2017
Mathew Shanley
Earlier today, Alynylam Pharmaceuticals announced it received Breakthrough Therapy Designation, granted by the U.S. Food and Drug Administration (FDA), for their porphyria treatment givosiran (ALN-AS1).
Givosiran is an investigational ribonucleic acid interference (RNAi) therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) to prophylactically reduce porphyria-related attacks in patients with acute hepatic porphyria (AHP). AHP is a form of porphyria in which the liver experiences enzyme deficiency, and includes acute intermittent porphyria (AIP), hereditary coproporphyria (HCP), and variegate porphyria (VP).
The Breakthrough Therapy designation is intended to assist in ensuring that patients with unmet medical needs can receive access to new therapies through FDA approval as quickly as possible.
“Patients with acute hepatic porphyrias, a family of ultra-rare diseases, suffer from severe neurovisceral attacks often resulting in hospitalization, and chronic, debilitating symptoms that impair daily function. This FDA decision is recognition of both the need for novel therapeutic options and the promising initial results with givosiran,” said Jeff Miller, Vice President, General Manager, Givosiran Program at Alnylam.
“We believe givosiran could become a transformative treatment for patients with this devastating and potentially life-threatening disease. Accordingly, we look forward to rapidly advancing this program in collaboration with global regulatory authorities, having also received PRIME designation from the European Medicines Agency earlier this year. We plan to initiate the Phase 3 clinical program with givosiran in late 2017.”
The ongoing Phase 1 study of givosiran has shown promising results and demonstrated significant reductions in the occurrence of porphyria-related attacks, leading to the Breakthrough application. The study is being conducted as randomized, double-blind and placebo-controlled trial, and data presented at the 2016 American Society of Hematology (ASH) meeting demonstrated initial evidence for clinical activity with the drug. During that meeting, preliminary data showed a 74% reduction in annualized attack rate.
AHP patients have been known to suffer from an array of symptoms that can include acute and/or recurrent life-threatening attacks with severe abdominal pain, peripheral and autonomic neuropathy, neuropsychiatric manifestations, cutaneous lesions and potentially paralysis and even death if not treated immediately.
During the 2016 ASH meeting, RDR spoke with Pushgal Garg, MD of Alnylam about givosiran for the treatment of AHP. That interview is shown below.

Updated results from this trial will be provided in an oral presentation on June 26, 2017 at the International Congress on Porphyrins and Porphyrias (ICPP) being held in Bordeaux, France.

Stay informed on the latest rare disease news and developments by signing up for our newsletter.
Copyright © RareDR 2013-2018 Rare Disease Communications. All Rights Reserved.