#Sarepta

@pharmalot.bsky.social

2 weeks ago

Pharmalittle: We're reading about a puzzling FDA rejection, a Lilly weight loss pill, and more An experimental therapy for a rare blood cancer was rejected by the FDA though internal reviewers had recommended it be cleared

Pharmalot.. Pharmalittle.. Good Morning: We’re reading about a puzzling FDA rejection, a Lilly weight loss pill, and more news.. statnews.com/pharmalot/20... #pharma #FDA #obesity #weightloss #HIV #Gilead #Sarepta #EliLilly

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pharmaphorum

@pharmaphorum.bsky.social

2 weeks ago

Changes at the top of Sarepta, Alkermes, as CEOs depart Two long-serving pharma CEOs have announced their departures, as Sarepta's Doug Ingram and Alkermes' Richard Pops announce their retirement.

Two long-serving #pharmachiefexecutives have announced their departures, as #DougIngram steps down from #Sarepta and #RichardPops plans his #retirement from #Alkermes.

pharmaphorum.com/news/changes...

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tickerade

@tickerade.bsky.social

2 months ago

Assessing Sarepta Therapeutics (SRPT) Valuation After a Recent Share Price Rebound Why Sarepta Therapeutics Stock Is Back on Investors Radar Sarepta Therapeutics (SRPT) has quietly bounced over the past month, even after a tough year where the stock is still down sharply. That disco...

Assessing #Sarepta Therapeutics, #SRPT, Valuation After a Recent Share Price Rebound

simplywall.st/stocks/us/ph...

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tickerade

@tickerade.bsky.social

3 months ago

Sarepta Therapeutics (NASDAQ:SRPT) Shares Up 5.3% - Still a Buy? Sarepta Therapeutics (NASDAQ:SRPT) Stock Price Up 5.3% - Still a Buy?

#Sarepta Therapeutics $SRPT Shares Up 5.3% - Still a Buy?

www.marketbeat.com/instant-aler...

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@jyves94.bsky.social

3 months ago

Dyne Therapeutics plans to file for approval for next-generation Duchenne drug Dyne Therapeutics plans to submit its next-gen Duchenne muscular dystrophy drug to the FDA after a successful trial.

⚡️ #Dyne Therapeutics prévoit de déposer une demande d’approbation pour la prochaine génération du médicament contre la dystrophie musculaire #Duchenne 💪 à la #FDA après un essai réussi

Le médicament pourrait être une version + efficace du controversé 💊 #Sarepta

www.statnews.com/2025/12/08/d...

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Alicia Ault

@aliciaault.bsky.social

3 months ago

Liver Deaths Prompt FDA Boxed Warning for Elevidys for DMD The FDA is adding a Boxed Warning label to delandistrogene moxeparvovec-rokl, a gene therapy for Duchenne muscular dystrophy, after reports of fatal liver injuries.

ICMYI: FDA issues new warning for Elevidys for Duchenne muscular dystrophy.

via @medscape.com

#Medsky #Neurosky #raredisease #Sarepta

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@pharmalot.bsky.social

3 months ago

Pharmalittle: We're reading about Novo plans for Wegovy, a Purdue bankruptcy deal, and more Novo Nordisk has begun selling Wegovy for $349 a month to cash payers, months ahead of the timeline set out with the Trump administration

Pharmalot.. Pharmalittle.. Good Morning: We’re reading about Novo plans for Wegovy, a Purdue bankruptcy deal, and more news.. statnews.com/pharmalot/20... #pharma #biotech #opioids #weight #obesity #FDA #NovoNordisk #JNJ #Purdue #Sarepta #musculardystrophy #bankruptcy

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@newtonianmade.bsky.social

4 months ago

Last night, Sarepta $SRPT announced 3rd quarter results. They were not good. A major decline in GTx revenue. They also announced that the confirmatory study for 2 PMOs was not positive after 96 wks. 3 PMOs confirmatory trials have now failed.

#sarepta

investorrelations.sarepta.com/news-release...

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@pharmalot.bsky.social

5 months ago

Pharmalittle: We're reading about Trump and BlinkRx conflicts, a setback for Sarepta, and more Pharma news of the day, as told by the Pharmalittle newsletter.

Pharmalot.. Pharmalittle.. Good Morning: We’re reading about Trump and BlinkRx conflicts, a setback for Sarepta & more news.. statnews.com/pharmalot/20... #pharma #Medicaid #medicines #UK #FDA #antitrust #Trump #India #Sarepta #Lilly #AstraZeneca

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@pharmalot.bsky.social

5 months ago

Sarepta suffers a setback as N.Y. panel recommends state Medicaid pause coverage of Duchenne drug In a setback for Sarepta Therapeutics, a New York panel has voted unanimously to recommend the state Medicaid program pause coverage of a treatment for Duchenne muscular dystrophy.

Sarepta suffers a setback as N.Y. panel recommends state Medicaid pause coverage of Duchenne drug.. statnews.com/pharmalot/20... #pharma #Medicaid #Sarepta #Duchenne #musculardystrophy #Elevidys #FDA

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@pharmalot.bsky.social

6 months ago

Pharmalittle: We're reading about Congress and drug pricing, questions on a Sarepta drug, and more In today's Pharmalittle roundup, we're reading about Congress eyeing a Trump drug pricing plan, questions on a Sarepta drug, and more.

Pharmalot.. Pharmalittle.. Good Morning: We’re reading about Congress and drug pricing, questions on a Sarepta drug & much more news.. statnews.com/pharmalot/20... #pharma #weight #obesity #UK #patents #musculardystrophy #novonordisk #lilly #sarepta

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Muscular Dystrophy Pakistan

@mdpakistanorg.bsky.social

7 months ago

Let's count down 30 September and mark your calendar...

Join our hands to make event successful.

#musculardystrophypakistan #livingwithlgmd #LGMD2A #MuscularDystrophyAwareness #sarepta #everylifefoundation

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Oninvest

@oninvest.bsky.social

7 months ago

Shares in biotech Sarepta soar after FDA allows shipments of Elevidys to resume – Oninvest Shares of Sarepta Therapeutics, a small-cap developer of genetic medicines to treat rare neuromuscular and central nervous system diseases, soared 14% yesterday, July 29. The driver of the gains was t...

#Sarepta soars as #FDA clears Elevidys for ambulatory DMD patients after ruling that a recent patient death was unrelated to the treatment.

While shipments for non-ambulatory patients remain paused, analysts say the decision removes a worst-case scenario.

More👇 en.oninvest.com/article/shar...

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@pharmalot.bsky.social

7 months ago

Pharmalittle: We're reading about a new Novo Nordisk CEO, an FDA reversal on Sarepta, and more Novo Nordisk tapped Maziar Mike Doustdar, its head of international operations, as its new chief executive officer and president

Pharmalot.. Pharmalittle.. Good Morning: We’re reading about a new Novo Nordisk CEO, an FDA reversal on Sarepta & more news.. statnews.com/pharmalot/20... #pharma #FDA #CDC #RFKJr #vaccines #pharmacies #Trump #tariffs #Sarepta #supermarkets

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pharmaphorum

@pharmaphorum.bsky.social

7 months ago

#Sarepta has restarted distribution of its #Duchenne muscular dystrophy gene therapy #Elevidys – but only for patients who can walk.

pharmaphorum.com/news/fda-giv...

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DMD WarrioR

@dmdwarrior.com

7 months ago

FDA: ‘Elevidys Gene Therapy Will Continue to Be Used in Ambulatory DMD Patients’ In a statement Monday, it said the FDA's swift action eliminated a problem with Sarepta, allowing Elevidys to return to the market without another safety study as had been feared.

FDA: ‘Elevidys Gene Therapy Will Continue to Be Used in Ambulatory DMD Patients’

#dmd #sarepta #roche #elevidys #fda #duchenne

👉 Read More: DMDWarrioR.com

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DMD WarrioR

@dmdwarrior.com

7 months ago

FDA Investigating Death of 8-Year-Old Brazilian Boy Who Received Elevidys The U.S. Food and Drug Administration is investigating the death of an 8-year-old boy who received Elevidys, a Sarepta Therapeutics gene therapy for Duchenne muscular dystrophy. The death occurred on June 7, 2025. The FDA has requested and voluntary suspension of product distribution as it investigates the safety concerns. The U.S. Food and Drug Administration (FDA) issued a press release announcing an investigation into the death of an eight-year-old Duchenne muscular dystrophy (Duchenne) patient who had received ELEVIDYS (delandistrogene moxeparvovec) gene therapy.

FDA Investigating Death of 8-Year-Old Brazilian Boy Who Received Elevidys

#dmd #sarepta #roche #elevidys #fda #duchenne

👉 Read More: DMDWarrioR.com

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DMD WarrioR

@dmdwarrior.com

7 months ago

The European Medicines Agency (EMA) Issued a Negative Opinion on Elevidys A European Medicines Agency committee ruled that the therapy, called Elevidys, failed to show in studies that it improved patients’ movement abilities. Roche announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) issued a negative opinion on the conditional marketing authorisation (CMA) for Elevidys (delandistrogene moxeparvovec) for ambulatory individuals aged three to seven years with Duchenne muscular dystrophy (DMD).

The European Medicines Agency (EMA) Issued a Negative Opinion on Elevidys

#dmd #sarepta #roche #elevidys #europe #ema

👉 Read More: DMDWarrioR.com

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Profit Trading USA

@profittradingusa.bsky.social

7 months ago

Sarepta stock falls after Elevidys fails to get backing by EU regulators Investing.com -- Sarepta Therapeutics Inc. (NASDAQ:SRPT) stock declined 13% Friday after European regulators recommended against approving Elevidys, the company’s gene therapy for Duchenne muscular dystrophy. The European Medicines Agency’s drug advisory committee (CHMP) rejected Elevidys for children aged 3 to 7 years who are able to walk, the agency announced Friday. The therapy, which is marketed outside the U.S. by Roche Holding AG (SIX:ROG), was seeking approval for treating the rare genetic disease that causes progressive muscle weakness. This setback follows closely on the heels of the U.S. Food and Drug Administration’s recent request for a pause on Elevidys use in the United States. Sarepta had already announced a temporary halt on U.S. shipments earlier this week. Roche has also paused shipments in jurisdictions that reference FDA approvals, further limiting the therapy’s market reach. The Swiss pharmaceutical giant had been responsible for commercializing the treatment in markets outside the U.S. after partnering with Sarepta. Duchenne muscular dystrophy is a rare genetic disorder that primarily affects boys and leads to progressive muscle deterioration. The condition is ultimately fatal, with patients typically experiencing serious mobility limitations and shortened lifespans. The dual regulatory setbacks in both the U.S. and European markets represent significant challenges for Sarepta’s commercialization strategy for Elevidys, which had been positioned as a breakthrough treatment option for the debilitating condition.

Click Subscribe #Sarepta #Elevidys #StockMarket #Investing #EURegulators

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DMD WarrioR

@dmdwarrior.com

7 months ago

Roche Suspends Shipments of Elevidys Gene Therapy Outside of the United States

Roche Suspends Shipments of Elevidys Gene Therapy Outside of the United States

#dmd #sarepta #roche #elevidys

👉 Read More: DMDWarrioR.com

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pharmaphorum

@pharmaphorum.bsky.social

7 months ago

Sarepta backs down and pauses Elevidys shipments The FDA seems to have won its staring contest with Sarepta over shipments of Elevidys, as the company backs down and agrees to a pause from today.

The #FDA appears to have won its staring contest with #Sarepta over shipments of #Elevidys, after the company blinked and agreed to the regulator's request to temporarily suspend shipments of the #Duchenne muscular dystrophy (DMD) #genetherapy.

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David Usharauli

@davidusharauli.bsky.social

7 months ago

Back then, one patient death was enough to shut down gene therapy research for years. Now, it took three deaths and firm FDA action to stop a company from continuing unfazed.

#biotech
#FDA
#sarepta

www.fiercebiotech.com/biotech/sare...

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Profit Trading USA

@profittradingusa.bsky.social

7 months ago

These Stocks Are Moving the Most Today: Sarepta, Block, Tesla, Verizon, and More - MSN These Stocks Are Moving the Most Today: Sarepta, Block, Tesla, Verizon, and More MSN

Click Subscribe #StockMarket #Investing #Tesla #Sarepta #FinanceNews

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Profit Trading USA

@profittradingusa.bsky.social

7 months ago

Sarepta stock falls after FDA places clinical hold on gene therapy trials Investing.com -- Sarepta Therapeutics Inc (NASDAQ:SRPT) stock tumbled 10% following the announcement that the U.S. Food and Drug Administration has placed a clinical hold on the company’s investigational gene therapy clinical trials for limb girdle muscular dystrophy (LGMD). The FDA’s decision affects multiple Sarepta LGMD programs, including SRP-9003, SRP-9004, SRP-6004, and SRP-9005. This comes just days after the company had already paused most of these programs as part of a strategic restructuring process, with the exception of SRP-9003. Additionally, the FDA has revoked the platform technology designation for Sarepta’s AAVrh74 platform technology that was previously granted on June 2, 2025. The company stated it intends to discuss with the FDA a potential pathway to submit a Biologics License Application seeking accelerated approval for SRP-9003 after the clinical hold is lifted. The clinical hold follows recent controversy reported by Reuters, where U.S. regulators asked Sarepta to voluntarily halt shipments of its Elevidys gene therapy after a muscular dystrophy patient who received a different, experimental treatment died. Sarepta declined to fully comply, continuing shipments to ambulatory patients while maintaining a halt for non-ambulatory patients implemented on June 15. Several analysts adjusted their outlook on Sarepta following the news. Piper Sandler analyst Biren Amin lowered the price target to $18.00 from $32.00 while maintaining a Neutral rating. Cantor Fitzgerald analyst Kristen Kluska reiterated a Neutral rating with a $14.00 price target. TD Cowen analyst Ritu Baral, who maintained a Hold rating and $28.00 price target, commented, "Patterns of lack of disclosure undermine SRPT’s credibility with DMD patient groups, the power behind prior approvals. While pt groups were aware of the patient deaths, they are, and have been for some time, frustrated with SRPT’s lack of disclosure on the overall safety of Elevidys, including cases of sudden cardiac death post-Tx as well as rates of severe liver events/ALI. SRPT’s arguments this past week on the relevance of LGMD events have further eroded confidence in their disclosure ethos. We strongly believe that patient group support behind SRPT has greatly eroded since winter." This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

Click Subscribe #Sarepta #FDA #GeneTherapy #ClinicalTrials #StockMarket

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pharmaphorum

@pharmaphorum.bsky.social

7 months ago

Sarepta resists as FDA seeks pause on Elevidys use Sarepta has defied an FDA request to halt all use of its gene therapy for Duchenne muscular dystrophy, creating an intriguing regulatory impasse.

#Sarepta has refused to suspend all dosing of its #genetherapy for #Duchennemusculardystrophy, Elevidys, defying a clinical hold request from the #FDA.

pharmaphorum.com/news/sarepta...

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Nasdaq

@nasdaq.activitypub.awakari.com.ap.brid.gy

7 months ago

StockWatch: Sarepta Shares Nosedive after LGMD Gene Therapy Patient Dies The death of a third patient tied to a Sarepta Therapeutics (NASDAQ: SRPT) gene therapy this year sent the company’s share...

#Drug #Discovery #dies #gene #LGMD #Nosedive #patient […]

[Original post on zephyrnet.com]

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Nasdaq

@nasdaq.activitypub.awakari.com.ap.brid.gy

7 months ago

StockWatch: Sarepta Shares Nosedive after LGMD Gene Therapy Patient Dies The death of a third patient tied to a Sarepta Therapeutics (NASDAQ: SRPT) gene therapy this year sent the company’s sh...

#Drug #Discovery #dies #gene #LGMD #Nosedive #patient #Sarepta […]

[Original post on zephyrnet.com]

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National Center for Health Research

@nc4hr.bsky.social

7 months ago

Exclusive: US FDA to ask Sarepta to stop shipments of gene therapy Elevidys, source says The U.S. Food and Drug Administration is planning to request Sarepta Therapeutics to voluntarily stop all shipments of its gene therapy, Elevidys, a source familiar with the matter told Reuters on Friday.

#FDA tells #Sarepta to stop selling #Elevidys #gene therapy due to deaths of 3 gene therapy patients. For years, the FDA kept approving Sarepta drugs costing millions per patient despite insufficient evidence. Read more: www.reuters.com/business/hea...

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Profit Trading USA

@profittradingusa.bsky.social

7 months ago