FDA grants orphan drug status to ALS gene therapy SNUG01
Sineugene planning clinical trial in China, US
The U.S. Food and Drug Administration (FDA) has granted orphan drug status to Sineugene Therapeutics‘ SNUG01, a gene therapy for people with amyotrophic lateral sclerosis (ALS).
The designation is given to potential treatments for rare diseases, or those affecting fewer than 200,000 people in the U.S. It provides the company with a range of development and commercial incentives, including tax credits for clinical trials, exemption from certain fees, and eligibility for seven years of marketing exclusivity, if approved.
The announcement follows Sineugene’s clearance by the FDA to initiate a global Phase 1/2a clinical trial to evaluate the safety, tolerability, and preliminary efficacy of SNUG01 in adults with ALS. The study plans to enroll patients at sites in China, as well as at Massachusetts General Hospital in the U.S., according to a company press release.
ALS is a rapidly progressive disorder characterized by the degeneration of nerve cells that control voluntary movements, called motor neurons, leading to gradually worsening muscle weakness.
While treatments are available to slow disease progression and improve survival, their benefits are limited, and more effective therapies are needed to improve outcomes for people with ALS.
Orphan drug status follows promising studies
Researchers at Tsinghua University in China discovered that a protein called TRIM72 had certain neuroprotective properties that made it a potential target for ALS gene therapy. In fact, boosting its production in nerve cells counteracted a number of disease mechanisms in ALS mouse models, leading to slower disease progression and improved survival.
Building on these findings, Sineugene developed SNUG01, a first-in-class gene therapy that’s designed to deliver the human TRIM72 gene to nerve cells. The gene is packaged inside a modified, harmless viral vector and is administered via a one-time injection into the spinal canal.
While most gene therapies in development for ALS target specific genetic mutations, meaning they are intended for a small subset of patients, SNUG01’s broad range of neuroprotective mechanisms could make it a promising treatment approach for more than 90% of ALS patients, according to Sineugene.
In an investigator-initiated trial conducted in China, SNUG01 showed early signs of effectiveness and a favorable safety and tolerability profile. SNUG01 treatment for more than 15 months in a compassionate use study (ChiCTR2400085764) also stabilized the disease in a patient whose disease had been rapidly progressing before treatment.
A second investigator-initiated trial (NCT06645197) was launched earlier this year to evaluate the safety and efficacy of different doses of SNUG01 in up to seven ALS patients at multiple sites in China.
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