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.

The post Pfizer & Sarepta Gene Therapies Both Failed Phase 3, But Analysts Expect Sarepta Will Win Approval appeared first on MedCity News.

The PTC Therapeutics drug Translarna failed its confirmatory study in Duchenne muscular dystrophy. Analysts say the European Commission’s decision to not adopt the Committee on Medicinal Products for Human Use’s negative opinion on the drug is unusual, if not unprecedented.

The post European Commission Decision Is a Reprieve for PTC Therapeutics’ Rare Muscle Disease Drug appeared first on MedCity News.

Corticosteroids are a standard first-line treatment for Duchenne muscular dystrophy, but these drugs have many side effects. FDA approval of Italfarmaco’s Duvyzat is the first nonsteroidal drug to pass the agency’s regulatory bar for treating this rare disease.

The post FDA Nod in Duchenne Helps Wider Swath of Patients With the Rare Muscle Disease appeared first on MedCity News.

Dyne Therapeutics recently reported encouraging Phase 1/2 clinical data in myotonic dystrophy type 1 and Duchenne muscular dystrophy. At the J.P. Morgan Healthcare Conference, CEO Joshua Brumm said Dyne’s existing cash combined with the new capital is expected to last through 2025—well beyond the next key milestones for both therapeutic candidates.

The post Genetic Medicines Biotech Dyne Pulls In $345M for Clinical Tests of Muscle Drugs appeared first on MedCity News.

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.

The post Sarepta Gene Therapy Misses in Phase 3; Prospects Now Rely on FDA Flexibility appeared first on MedCity News.

Rich Horgan believes that there’s an alternative path to drug development that can lower costs and the time it takes to bring drugs to market. He is trying to apply it to the field of rare and ultra-rare diseases.

The post MedCity Pivot Podcast: Tackling Rare Disease With Someone Who Was Touched By It appeared first on MedCity News.

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.

The post FDA Approves First Gene Therapy for Duchenne Muscular Dystrophy appeared first on MedCity News.

Private equity firm KKR teamed up with OMX Ventures to lead the seed investment in Replay, a company developing a suite of technologies that could overcome capacity limitations of adeno-associated viruses used for genetic medicines delivery. Duchenne muscular dystrophy is among the disease targets of the new startup.

The post Startup aiming to push boundaries of gene therapy nets $55M in seed cash appeared first on MedCity News.

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.

The post With new gene therapy data in hand, Sarepta talks with FDA about approval pathways appeared first on MedCity News.

A Duchenne muscular dystrophy drug candidate from Italfarmaco Group has encouraging preliminary data from a pivotal study. The Milan, Italy-based pharmaceutical company says it now plans to meet with U.S. and European authorities about seeking regulatory approval for the small molecule.

The post As muscular dystrophy drug meets key goal, Italfarmaco plans march forward to regulators appeared first on MedCity News.

Code Biotherapeutics uses synthetic DNA as the foundation for its genetic medicines, which the startup claims offer several key advantages compared to genetic medicines delivered via engineered viruses. The startup plans to use its new capital to develop lead programs in Duchenne muscular dystrophy and type 1 diabetes.

The post Code Bio corrals $75M to skip viruses, use synthetic DNA for genetic meds delivery appeared first on MedCity News.

PepGen’s technology improves the delivery of a therapy to more tissue types and the clinical-stage biotech plans to use its IPO cash to continue developing its lead program for Duchenne muscular dystrophy. Meanwhile, eye products giant Bausch + Lomb returned to the public markets as a standalone company. Both the PepGen and Bausch + Lomb IPOs priced below their respective price ranges.

The post PepGen IPO nabs $108M for muscular dystrophy drug with potential edge over Sarepta med appeared first on MedCity News.

Pfizer now has the regulatory O.K. to resume pivotal tests of its gene therapy for Duchenne muscular dystrophy—but with new limitations and safety measures. The FDA had placed a clinical hold on the study late last year following a patient death.

The post FDA lifts Pfizer gene therapy clinical hold, but trial adds new safety measures appeared first on MedCity News.

The FDA has halted tests of a Pfizer gene therapy for Duchenne muscular dystrophy. The clinical hold is one of several placed on gene therapy tests in the past year due to safety concerns.

The post Patient death prompts FDA halt on Pfizer’s Duchenne gene therapy study appeared first on MedCity News.

Capricor Therapeutics’ cell therapy for Duchenne muscular dystrophy posted encouraging results from a Phase 2 study enrolling 20 patients. But the FDA said a Phase 3 test is needed to support a regulatory submission, and the biotech is hunting for a pharmaceutical industry partner.

The post Capricor’s muscle drug data look good, but partner wanted and Phase 3 test needed appeared first on MedCity News.

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.

The post FDA approves Sarepta drug for muscular dystrophy with rare genetic mutation appeared first on MedCity News.

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.

The post Documents give inside view of FDA’s rejection, and surprise approval, of Sarepta Duchenne’s drug appeared first on MedCity News.

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.

The post Roche, Sarepta Therapeutics sign $1.15B deal for ex-US rights to gene therapy for Duchenne’s appeared first on MedCity News.

The FDA had previously rejected Sarepta’s application for Vyondys 53 in August, citing infections and kidney toxicity in preclinical studies. However, some analysts saw the move as a ‘slap on the wrist’ for the controversial approval of Sarepta’s first DMD drug.

The post Sarepta wins surprise approval for second Duchenne muscular dystrophy drug appeared first on MedCity News.

In a note to investors, SVB Leerink analyst Joseph Schwartz suggested that the ‘questionable’ approval of Sarepta’s Exondys 51 may have played a role in the FDA’s decision to turn down golodirsen.

The post FDA rejection of Sarepta drug may be a ‘slap on the wrist’ over earlier approval, analyst writes appeared first on MedCity News.

The decision stood in contrast with the agency’s highly controversial approval of Sarepta’s previous DMD drug, Exondys 51. The company’s shares fell sharply in after-hours trading.

The post FDA gives thumbs down to Sarepta’s next Duchenne’s muscular dystrophy drug appeared first on MedCity News.

Data from the drugmaker’s Phase Ib study showed patients responding, but an analyst wrote that it appears less potent and to have higher toxicity than that of its competitor, Sarepta Therapeutics.

The post Pfizer gene therapy shows early efficacy in Duchenne muscular dystrophy, but data raise questions appeared first on MedCity News.

An analyst wrote that the therapy’s combination of AAV delivery with a validated means of treating DMD could distinguish it from other gene therapies in what is already a crowded space.

The post Audentes, Nationwide Children’s to develop gene therapy in Duchenne muscular dystrophy appeared first on MedCity News.

After two painfully incremental drug approvals, the Duchenne muscular dystrophy field is still searching for a therapy that can substantially improve patients’ lives and lifespan. Sounds like a job for CRISPR/Cas9.

The post Can CRISPR deliver real progress in Duchenne muscular dystrophy? appeared first on MedCity News.

Emflaza, the brand name for deflazacort, has never been approved for sale in the United States, but the steroid has been sold for decades in other countries — at much lower prices

The post 5 reasons for the latest drug cost firestorm, this time over Emflaza’s price tag appeared first on MedCity News.

Marathon Pharmaceuticals is doing some backtracking after the public, the media and now Bernie Sanders objected to the list price of its upcycled corticosteroid for Duchenne muscular dystrophy.

The post Four days later, Marathon postpones launch of $89K corticosteroid appeared first on MedCity News.

The FDA has given the green light to Emflaza (deflazacort), a new drug for Duchenne muscular dystrophy. Announced Thursday, the news is bittersweet. Deflazacort is a corticosteroid, a class of drugs that doctors have used off-label for many years.

The post FDA approves Duchenne muscular dystrophy drug appeared first on MedCity News.

Exondys 51 doesn’t cure Duchenne muscular dystrophy and will only help a minority of patients.

The post FDA greenlights Sarepta Therapeutics’ milestone treatment for Duchenne muscular dystrophy appeared first on MedCity News.

The post Morning Read: BioMarin pulls plug on $680M Duchenne drug, Valeant’s Pearson cashes in appeared first on MedCity News.

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