FDA's Approval For Sarepta's Duchenne Gene Therapy, A Boost for Taysha Gene's Rare Neuropathy Treatment: Analyst Weighs

On Friday, the FDA's adcomm voted 8 to 6 in support of accelerated approval of Sarepta Therapeutics Inc's SRPT SRP-9001 (delandistrogene moxeparvovec) for ambulatory patients with Duchenne muscular dystrophy.

Truist analyst writes that given the controversial dataset with an equally controversial mechanism of action of SRPT-9001, the vote comes as a surprise. However, despite being narrow, it is positive for Taysha Gene Therapies Inc's TSHA TSHA-120. 

The analyst has raised the price target to $3 from $2 with a Buy rating.

It says that the FDA would eventually recognize that a placebo-controlled study of TSHA-120 for giant axonal neuropathy (GAN) is not feasible due to the ultra-rare nature of the disease and accept natural history as a reasonable comparator. 

The replacement of the full copy of the GAN gene, gigaxonin, used in TSHA-120 has a stronger rationale than the controversial micro-dystrophin for DMD. 

The analyst also notes that TSHA-120's safety and efficacy data is impressive compared to the limited dataset available for SRPT-9001.

The formal FDA meeting request submission to discuss the regulatory path forward for TSHA-120 in GAN is expected in Q2 2023; the formal meeting is anticipated in Q3 2023.

Price Action: TSHA shares are up 3.44% at $0.69 on the last check Monday.