| | | |   | Hi friends, it's Meghana. Today, we dive deep into the current status of sickle cell drug development, and why so few patients are receiving these therapies. Also up for discussion: The ongoing shortages of several diabetes drugs. | | | Sickle cell drugs are available — but are they accessible? There’s an amazing array of modern therapies for sickle cell disease. There are the new gene therapies, of course, meant to correct the underlying mutations that cause the disease — and then there are pills like Oxbryta that prevent blood cells from convoluting into a sickle shape. But only a fraction of patients eligible for these potent treatments are actually taking them, STAT’s Jason Mast writes. That may be because sickle cell predominantly affects Black people, who face steep inequities in health care. The red tape involved in getting insurers to approve Oxbryta, which retails for around $125,000 a year, poses another major obstacle for sickle cell patients. That said, drugmakers are investing billions in developing even more treatments for sickle cell disease. The current gene therapies are unlikely to be given to patients outside the U.S. and Europe, while most of the world’s 120 million sickle cell patients live in South Asia and West Africa. A pill could have global reach, STAT’s Jason Mast writes. Read more. And more. | Biopharma CEO of the Year! It’s been a rough year for deal-making in biopharma, but there are still some deserving contenders in the running for the sector’s top CEO of 2022, STAT’s Adam Feuerstein writes. Tim Walbert of Horizon Pharmaceuticals gets a nod for forging that $27.8 billion deal with Amgen, among other accomplishments. And Vertex Pharmaceuticals’ Reshma Kewalramani gets an honorable mention for developing a uniquely exciting research pipeline. But the top award goes to Haruo Naito of Eisai, which has reinvigorated the Alzheimer’s field with the positive performance of lecanemab this year. The execution of the drug’s development was practically flawless, and the likely prospect of an FDA approval for lecanemab has raised the hopes of millions in the Alzheimer’s community. Read more. | | | A dearth of diabetes drug Mounjaro There’s a shortage of the Eli Lilly diabetes drug Mounjaro, the FDA says. The demand for Mounjaro is mounting, meaning that shipments of the drug could be delayed through January. Drugs like Mounjaro, Lilly’s Trulicity, and Ozempic, made by Novo Nordisk, have seen a dramatic spike in popularity after gaining popularity on social media for their weight-loss effects. Now, some pharmacies are restricting the drug to patients who have confirmed diabetes cases before filling prescriptions — and Lilly itself is limiting its savings coupons to people with type 2 diabetes. Lilly plans on doubling production of the drug, which should kick off in the middle of next year at a North Carolina facility. Read more. | Psychedelics without the trip Drugmakers are increasingly trying to take the “high” out of psychedelic substances so that mind-bending drugs like MDMA, LSD, and psilocybin can be used therapeutically for a broader array of patients. These drugs are being studied as treatments for mental health conditions like depression, PTSD, and addiction. “We have to create medicines that are safe enough that people can take them at home and put them in their medicine cabinet,” an executive at Delix Therapeutics told the Wall Street Journal. That company has raised about $100 million, and is moving two drugs — which are psychedelics without the trip — into human trials next year. Many more are following suit. | Coherus has its PDUFA date on Friday Coherus BioSciences said in July that the FDA would be reviewing a biologics license application for its cancer drug, toripalimab, with a PDUFA date coming right in time for the holidays on December 23. The company is looking to have toripalimab approved for metastatic nasopharyngeal carcinoma, when combined with chemotherapy. Last year, the agency initially accepted a BLA for the antibody drug, but in May 2022 the FDA issued a complete response letter asking Coherus for a quality process change. Data for the old and resubmitted BLAs come from a Phase 2 trial, which had an objective response of 20.5% with toripalimab alone, and from a Phase 3 trial, which had a median progression-free survival of 11.7 months when toripalimab was combined with gemcitabine and cisplatin. Placebo had a PFS of 8.0 months. If the drug is approved, Coherus plans on launching toripalimab in the U.S. in the first quarter of 2023. | More reads - After prior rejection, Ferring finally nabs FDA nod for bladder cancer gene therapy, FiercePharma
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| Thanks for reading! Until tomorrow,  | | | |
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