| | | |   | Morning — it’s Meghana. We’ve got a new gene therapy approval, a debate over the merits of three controversial cancer drugs, and more. | | | ODAC to discuss three drugs’ fate this week The FDA’s Oncologic Drugs Advisory Committee will meet on Thursday and Friday this week to discuss two accelerated approvals that are now in doubt, and another controversial pill.
On the docket is Oncopeptides’ Pepaxto, which was granted accelerated approval last year for relapsed or refractory multiple myeloma. Since then, a confirmatory study showed that it missed its overall survival endpoint and had a higher risk of death than Bristol Myers Squibb’s Pomalyst. The company toyed with pulling Pepaxto from the market, but has yet to do so. The ODAC will also weigh the benefits of Secura Bio’s PI3K inhibitor, Copiktra. According to the FDA, the drug could increase the risk of death and has severe side effects for patients with certain blood cancers.
Lastly, the panel will discuss Spectrum Pharmaceuticals’ new drug application for its lung cancer medicine, poziotinib. Spectrum's efforts to receive a “breakthrough” designation for the drug were rejected in 2018, but newer Phase 2 data show some nominal promise. Poziotinib’s PDUFA date is Nov. 24. | Why the slowdown in Covid drug development? Drugmakers were initially able to develop Covid treatments at lightning speed: Between January 2020 to February 2021, four different and effective therapies were developed and deployed in hospitalized Covid patients. That pace has slowed considerably: In the past year-and-a-half, no new therapies have emerged, though a need for them remains. The stalling is at least partly due to the progress made on other Covid fronts. Back in 2020, so many people were hospitalized that studies were able to be conducted on a large scale, and results analyzed in real time. But the new vaccines and antivirals reduced the odds that patients would be hospitalized with Covid or die from it. That doesn't mean there's not still a need. “I was on service last week and had six critical Covid patients, and three of them died,” one Vanderbilt physician told STAT's Jason Mast. “I just got a text five minutes ago that a friend’s husband died of Covid yesterday. He was 58. People think it’s not happening, but it’s absolutely still happening.” Read more. | Can biopharma bring innovative novel therapeutics to market faster? As biopharma companies chart a course for innovative novel therapeutics, they often struggle to find the balance between speed to market and quality control. How do manufacturers effectively develop new therapy modalities while working within the confines of an established bioprocessing workflow? Cytiva can help lead the way. Find out how we work with scientists to advance and accelerate novel therapies. | Another gene therapy approval for Bluebird Bio Bluebird Bio has achieved its second regulatory win in the past month: The FDA has granted accelerated approval for the company’s personalized gene therapy for cerebral adrenoleukodystrophy, or CALD, an extremely rare and lethal childhood brain disease. Last month, Bluebird’s beta thalassemia gene therapy was also green-lighted. Expert panels voted unanimously to clear both treatments, despite some concern that they may cause cancer. The newly approved treatment, called Skysona, is made from a patient’s own stem cells, and will cost $3 million per patient, the Boston Globe's Ryan Cross reports. It’s estimated that about 50 boys are born each year in the U.S. that will eventually develop CALD. Bluebird expects to treat about 10 each year. The disease is caused by a single genetic mutation that impacts how cells metabolize certain fatty acids, leading them to accumulate in the central nervous system and blood.
Read more. | A new leader for top drug pricing group Patients for Affordable Drugs, one of Washington’s most prominent advocacy groups tackling high drug prices, has a passionate new leader. Merith Basey, who most recently led a group that pressures universities to improve patient access to medicine, has also advocated for equitable access to Covid-19 vaccines, tests, and treatments. She worked along with young people with type 1 diabetes in Ecuador, and co-founded the 100 Campaign, which worked to frame insulin access as a human rights issue. “Our number one challenge will be that the pharmaceutical industry is not going to quit overnight,” Basey told STAT's Rachel Cohrs. “They’ve spent hundreds of millions of dollars to prevent Medicare negotiation, and we know that Patients for Affordable Drugs won’t be stopping either.” Read more. | More reads - Lawsuit claims Pfizer fellowship is biased against whites, Asian-Americans, Reuters
- Intellia says CRISPR treatment safely corrects DNA of six patients with rare disease, STAT
- Ahead of FDA, EMA gives thumbs-up to AstraZeneca, Sanofi’s RSV antibody for infants and newborns, Endpoints
| Thanks for reading! Until tomorrow,  | | | |
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