Category: rare diseases
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STAT+: The FDA, urged to avoid controversy, creates a new headache with attack against UniQure
STAT+: The FDA, urged to avoid controversy, creates a new headache with attack against UniQure There was a telling exchange during a Thursday media call set up by the Food and Drug Administration to enable a “senior FDA official” — he could only be quoted anonymously — to attack UniQure and its experimental treatment for…
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STAT+: A rare disease drug was approvable, then it wasn’t. Inside a surprise rejection by the FDA
STAT+: A rare disease drug was approvable, then it wasn’t. Inside a surprise rejection by the FDA An experimental therapy for a rare blood cancer was on the path toward approval by the Food and Drug Administration last year — with internal reviewers recommending it be cleared — before the agency rejected the drug last…
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STAT+: FDA unveils rules for bespoke gene therapies, predicting flood of rare disease applications
STAT+: FDA unveils rules for bespoke gene therapies, predicting flood of rare disease applications The Trump administration on Monday released detailed guidance for approving the first bespoke medicines crafted to treat patients’ individual mutations. Food and Drug Administration Commissioner Marty Makary and biologics chief Vinay Prasad had already previewed the new approach, known as the plausible mechanism…
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STAT+: FDA rejects Regenxbio’s rare-disease gene therapy
STAT+: FDA rejects Regenxbio’s rare-disease gene therapy The Food and Drug Administration has rejected a rare-disease gene therapy from Regenxbio, the company said Monday. The one-time treatment, called RGX-121, is designed to replace a malfunctioning gene that causes mucopolysaccharidosis type II, also known as Hunter syndrome, an ultra-rare disorder that causes physical and cognitive impairments. …
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STAT+: FDA puts off Stoke’s request for faster filing of severe epilepsy drug
STAT+: FDA puts off Stoke’s request for faster filing of severe epilepsy drug SAN FRANCISCO — Stoke Therapeutics and the Food and Drug Administration were unable to reach agreement on an expedited submission for the company’s severe epilepsy treatment, the company said Sunday. Following a meeting in December, the FDA did not shut the door…
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STAT+: Key scientist from Baby KJ team launches startup to scale personalized CRISPR medicines
STAT+: Key scientist from Baby KJ team launches startup to scale personalized CRISPR medicines In between bites of thin, crispy slices of pizza at Berkeley’s Pizzeria da Laura, gene editing researcher Fyodor Urnov and venture capitalist Johnny Hu began hatching a plan for a different kind of CRISPR company. It was spring 2024 and Urnov,…
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STAT+: A new drug allowed them to go in the sun for the first time. They’re terrified they may have to give it up
STAT+: A new drug allowed them to go in the sun for the first time. They’re terrified they may have to give it up It was particularly cold and windy for a November day, but that didn’t matter to Ginger Gonsalves and Becky Kelliher. The sun was out, and no one counted the seconds in…
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STAT+: FDA’s stronger warning on Sarepta gene therapy raises new questions about heart risk
STAT+: FDA’s stronger warning on Sarepta gene therapy raises new questions about heart risk New warnings and restrictions placed on Sarepta Therapeutics’ gene therapy last week by the Food and Drug Administration have cast a spotlight on an important but overlooked heart-safety risk. “Acute, serious, and life-threatening” cases of heart inflammation called myocarditis and elevations…
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STAT+: Sarepta to seek approval for gene therapy in rare form of muscular dystrophy
STAT+: Sarepta to seek approval for gene therapy in rare form of muscular dystrophy An experimental gene therapy from Sarepta Therapeutics increased levels of the gene missing in an ultra-rare form of muscular dystrophy, according to data the company presented Friday. The company has said it plans to file for approval in the disease, known…
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Party in biotech land and AstraZeneca’s miscalculation
Party in biotech land and AstraZeneca’s miscalculation Where did former vaccine regulator Peter Marks find a new job? How did a math error cost AstraZeneca a rare disease candidate? And is biotech back? We discuss all that on this week’s episode of the “The Readout LOUD,” STAT’s biotech podcast. We bring on Mizuho health care…
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A gene therapy success in Huntington’s and Trump’s autism announcement
A gene therapy success in Huntington’s and Trump’s autism announcement Why were doctors and investors so excited about trial results of UniQure’s gene therapy for Huntington’s disease? Are autism rates actually rising? And what does the research tell us about Tylenol and autism? We discuss all that on this week’s episode of the “The Readout…
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STAT+: Crinetics wins FDA approval for acromegaly drug, faces competition with Novartis and Ipsen
STAT+: Crinetics wins FDA approval for acromegaly drug, faces competition with Novartis and Ipsen SAN DIEGO — Crinetics Pharmaceuticals won Food and Drug Administration approval on Thursday for a drug for a rare and debilitating hormonal disorder — the company’s first approved product. But the treatment faces competition from pharma giants with blockbuster medicines already…
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STAT+: FDA permits use of Sarepta Therapeutics’ Duchenne therapy in younger patients after short-lived halt
STAT+: FDA permits use of Sarepta Therapeutics’ Duchenne therapy in younger patients after short-lived halt In a sharp reversal, the Food and Drug Administration on Monday said it was clearing the way for Sarepta Therapeutics to resume shipments of its gene therapy for Duchenne muscular dystrophy to some patients. The therapy, called Elevidys, will once…
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STAT+: Sarepta Therapeutics’ Duchenne therapy faces ‘arduous’ path back to market, senior FDA official says
STAT+: Sarepta Therapeutics’ Duchenne therapy faces ‘arduous’ path back to market, senior FDA official says Sarepta Therapeutics, the maker of a gene therapy for Duchenne muscular dystrophy that is being temporarily shelved because of safety concerns, faces an “arduous and treacherous path” to try to get it back onto the market, a senior Food and…
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STAT+: The crisis over Sarepta’s Duchenne therapy is a mess. But the lesson is clear
STAT+: The crisis over Sarepta’s Duchenne therapy is a mess. But the lesson is clear In a matter of a few days last week, Sarepta Therapeutics announced major layoffs, acknowledged the death of another patient receiving one of its gene therapies, and was urged by the Food and Drug Administration to suspend shipments of a…
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STAT+: Sarepta Therapeutics crisis is huge blow to Duchenne families, company
STAT+: Sarepta Therapeutics crisis is huge blow to Duchenne families, company Sarepta Therapeutics confronted one of the most serious crises in its history on Friday, as the Food and Drug Administration prepared to ask the company to halt all shipments of its gene therapy for Duchenne muscular dystrophy — a treatment that had been a…
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Opinion: We are mothers of Duchenne patients. Recent setbacks with Sarepta must not stop progress
Opinion: We are mothers of Duchenne patients. Recent setbacks with Sarepta must not stop progress They say death is one of life’s few certainties. For a boy or young man living with Duchenne muscular dystrophy, that certainty has a cruel twist: the anticipation of dying young. As mothers of children with this disease, we have…
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Inside an FDA drug rejection, and layoffs at Sarepta
Inside an FDA drug rejection, and layoffs at Sarepta Just how many employees is Sarepta Therapeutics laying off? And why did the Food and Drug Administration reject Ultragenyx’s rare disease drug over manufacturing qualms? We discuss all that and more on this week’s episode of “The Readout LOUD”. Read the rest… Allison DeAngelis, Adam Feuerstein,…
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STAT+: Sarepta Therapeutics lays off one-third of workforce in drastic cost-cutting move
STAT+: Sarepta Therapeutics lays off one-third of workforce in drastic cost-cutting move Sarepta Therapeutics laid off more than one-third of its workforce, or approximately 500 employees, the company announced Wednesday — a drastic cost-cutting move following the deaths of two teenagers that forced the company to restrict usage of its gene therapy for Duchenne muscular…
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STAT+: What’s next for Sarepta Therapeutics with gene therapy under fire?
STAT+: What’s next for Sarepta Therapeutics with gene therapy under fire? This story is an excerpt from Adam’s Biotech Scorecard, a subscriber-only newsletter. STAT+ subscribers can sign up here to get it delivered to their inbox. The Duchenne muscular dystrophy community is reeling following the second death of a patient from liver failure caused by Sarepta Therapeutics’…
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Opinion: Following second patient death, Duchenne muscular dystrophy families deserve answers about Elevidys
Opinion: Following second patient death, Duchenne muscular dystrophy families deserve answers about Elevidys On Sunday at 1 a.m. Eastern time, an announcement went out from the first-generation gene therapy company for Duchenne muscular dystrophy: A second patient who had been treated with Elevidys has died. This second family said yes to hope, yes to science,…
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Opinion: STAT+: The World Health Assembly’s giant leap on rare diseases
Opinion: STAT+: The World Health Assembly’s giant leap on rare diseases On May 24, the World Health Assembly, the governing body of the WHO, ratified landmark legislation that, if implemented fully, will change the future for people living with rare diseases. It calls for concrete action plans that will have far-reaching impacts across health care…
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STAT+: Day 2 of ASGCT: Good news for Baby KJ and the dream of personalized gene editing
STAT+: Day 2 of ASGCT: Good news for Baby KJ and the dream of personalized gene editing NEW ORLEANS — Greetings from ASGCT, where the gene therapy field had some great news for once, about a highly photogenic infant. Baby KJ gets a custom CRISPR edit to his genome Kiran Musunuru, a researcher at the University of…
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Prenatal genetic therapy for SMA shows promise in a mouse study
Prenatal genetic therapy for SMA shows promise in a mouse study Researchers have taken preliminary steps toward treating a devastating neurodevelopmental disorder, spinal muscular atrophy, with a genetic therapy delivered in the womb, before the worst damage to motor neurons can occur. The scientists at the University of California, San Francisco, and Johns Hopkins University…