Sarepta Therapeutics

Despite Phase 3 Failure, Sarepta Still Sees Path for Muscle Disease Drugs to Secure Full FDA Approval

Sarepta Therapeutics attributed the Phase 3 failure to the Covid-19 pandemic, during which many patients missed multiple doses of its Duchenne muscular dystrophy therapies. The company plans to discuss with the FDA traditional regulatory approvals based on the totality of data that includes real world evidence from the years these drugs have been commercially available under accelerated approvals.

By Frank Vinluan

BioPharma

Sarepta’s Future in Gene Therapy Remains Clouded, But Data From Partner Hansa Is a Bright Spot

Hansa Biopharma has encouraging early clinical results showing its drug imlifidase, used as a pretreatment, enabled Duchenne muscular dystrophy patients to receive the gene therapy Elevidys. Sarepta is searching for ways to mitigate excessive immune responses to Elevidys that can lead to serious and potentially fatal liver injury.

By Frank Vinluan

NEMT Partner Guide: Why Payers and Providers Should Choose MediDrive’s TMS

Alan Murray on improving access for medical transportation.

By MedCity News

Hospitals, Startups, Health Tech, Payers Providers,

Healthcare Moves: A Monthly Summary of Hires, Exits and Layoffs

July has seen a slew of executive hires, exits and layoffs across the healthcare sector. For example, Aledade and Teladoc Health welcomed new executives, and DexCom and Community Health Systems announced that their CEOs are departing. There were also layoffs at organizations including Best Buy Health, the VA and Sarepta Therapeutics.

By Katie Adams

Pharma BioPharma,

Sarepta CEO: ‘We Have, I Believe, a Very Laudable History of Being Extraordinarily Transparent’

Sarepta Therapeutics knew about the death of a patient treated with its experimental gene therapy for a type of limb-girdle muscular dystrophy, but said nothing publicly for a month. Financial analysts lambasted Sarepta executives for failing to disclose the fatality when the company announced a restructuring that includes stopping work on this limb-girdle program.

By Frank Vinluan

BioPharma, Pharma

Sarepta Slashes Staff, Maps Path Forward for Gene Therapy and Narrower Pipeline

Sarepta’s Elevidys will remain on the market with a black box warning. But this gene therapy for Duchenne muscular dystrophy still faces commercial headwinds and analysts say the company has work to do to win over patients and investors.

By Frank Vinluan

BioPharma, Pharma

Sarepta Stops Stop Shipments of Muscular Dystrophy Gene Therapy After Second Patient Death

Like the first death reported in March, Sarepta Therapeutics attributed the fatality to a case of acute liver failure following dosing of the Duchenne muscular dystrophy gene therapy, Elevidys. The company has proposed an immunosuppressive regimen to mitigate the liver injury risk posed by the therapy.

By Frank Vinluan

BioPharma

Liver Failure-Associated Death Reported in Patient Treated With Sarepta Gene Therapy Elevidys

Sarepta Therapeutics said the Duchenne muscular dystrophy patient recently had a cytomegalovirus infection, which can damage the liver and may have contributed to the fatality. It’s the first patient death out of more than 800 patients treated with Elevidys, a gene therapy for the rare muscle-wasting disease.

By Frank Vinluan

BioPharma, Pharma

FDA Approves Sarepta Gene Therapy, But Only After Top FDA Official Bucks Agency Staff

FDA reviewers concluded the data for Sarepta Therapeutics’ Elevidys were insufficient to show efficacy in the rare muscle disorder Duchenne muscular dystrophy. Peter Marks, the agency’s top biologics official, reviewed the same data and reached a different conclusion.

By Frank Vinluan

BioPharma, Pharma

Pfizer & Sarepta Gene Therapies Both Failed Phase 3, But Analysts Expect Sarepta Will Win Approval

A Pfizer gene therapy for Duchenne muscular dystrophy failed its Phase 3 clinical trial. But analysts draw distinctions between that therapy and Sarepta Therapeutics’ Elevidys, which awaits an FDA decision that could grant it full regulatory approval.

By Frank Vinluan

BioPharma, Pharma

Sarepta Gene Therapy Misses in Phase 3; Prospects Now Rely on FDA Flexibility

Sarepta Therapeutics’ Duchenne muscular dystrophy gene therapy fell short of its main Phase 3 goal, but the firm contends the full body of evidence supports expanding the therapy’s label to all patients who have the muscle-wasting disease. Analysts say prospects of the therapy, Elevidys, rest on FDA willingness to exercise flexibility it has already shown to rare disease drugmakers, including Sarepta.

By Frank Vinluan

BioPharma, Pharma

FDA Approves First Gene Therapy for Duchenne Muscular Dystrophy

Sarepta Therapeutics’ Elevidys is now the first FDA-approved gene therapy for Duchenne muscular dystrophy. Elevidys is an engineered version of a gene intended to restore function lost to the mutation at the root of the inherited muscle disease.

By Frank Vinluan

BioPharma, Pharma

With new gene therapy data in hand, Sarepta talks with FDA about approval pathways

Sarepta Therapeutics has more clinical data showing the safety and efficacy of its experimental gene therapy for Duchenne muscular dystrophy. A pivotal Phase 3 test is already underway and could post data next year but the company is also talking with regulators about the possibility of a submission under the accelerated approval pathway.

By Frank Vinluan

BioPharma

FDA approves Sarepta drug for muscular dystrophy with rare genetic mutation

The FDA decision gives Sarepta Therapeutics its third approved drug for Duchenne muscular dystrophy. The accelerated approval requires the biotech to conduct additional clinical testing to confirm the drug’s benefit.

By Frank Vinluan

BioPharma, Policy

Documents give inside view of FDA’s rejection, and surprise approval, of Sarepta Duchenne’s drug

Despite a strongly critical complete response letter - which the FDA sent in August but did not make public until Tuesday - the drugmaker successfully appealed and won approval for the drug, Vyondys 53, in December, to the surprise of many.

By Alaric DeArment

BioPharma

Roche, Sarepta Therapeutics sign $1.15B deal for ex-US rights to gene therapy for Duchenne’s

An analyst wrote that the deal would create value for Sarepta, which has a gene therapy in Phase II development for Duchenne muscular dystrophy, SRP-9001, but lacks the resources to commercialize it abroad.

By Alaric DeArment