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• Polycythemia vera, a rare blood disease leading to a range of cardiovascular problems, has a new FDA-approved treatment, a drug (rusfertide) whose first-in-class approach could make the therapy a new first-line treatment for this condition.

• The regulatory decision announced after last Friday’s market close covers the treatment of erythrocytosis in adults with polycythemia vera.

• Standard polycythemia vera treatment has been therapeutic phlebotomy, a procedure in which some of a patient’s blood is removed to bring the red blood cell count closer to normal levels.

• Mimrylo, which was initially developed by Protagonist Therapeutics, works in a way that more closely mimics the way the body manages erythrocytosis.

• The peptide drug is an engineered version of hepcidin, a hormone produced by the body. Naturally occurring hepcidin regulates iron in the body. By making less iron available to bone marrow, hepcidin reduces the amount of iron that can be used to make red blood cells.(MedCity News)

• The FDA approved a once-daily HIV pill that could help simplify care for some patients whose virus is already under control but who remain on complicated treatment regimens.

• The tablet combines bictegravir, the backbone of Gilead’s blockbuster HIV pill Biktarvy, with lenacapavir, a centerpiece of the company’s long-term strategy for HIV treatment and prevention.

• The new pill is also aimed at people who are doing well on a single-tablet regimen, including Biktarvy, and want to switch to a new treatment.

• The approval makes the new pill the first single-tablet regimen available for adults with HIV whose virus is suppressed but are unable to use currently available one-pill treatment options, according to Gilead. That population accounts for an estimated 5% or more of individuals in the US living with HIV, the company said. (CNBC)

• In their high-profile competition in the diabetes and obesity markets, Eli Lilly has proven adept at playing catch-up with Novo Nordisk.

• On Friday, Lilly checked another item off that list as it secured FDA approval for Mounjaro to reduce the risk of major adverse cardiovascular events (MACE), including CV death, heart attack or stroke, in at-risk adults with Type 2 diabetes.

• With the nod, Lilly matches the 2020 approval Novo received for its diabetes treatment, Ozempic, to lower the risk of adverse cardiovascular events in at-risk patients. (Fierce Pharma)

• Novartis and Bristol Myers Squibb have paused multiple trials evaluating their respective cell therapy programs against autoimmune conditions after observing inflammatory side effects in testing, the companies confirmed Monday afternoon.

• Novartis, which is testing a personalized cell therapy called rap-cel in a handful of autoimmune studies, found three cases of immune effector cell-associated hemophagocytic syndrome, a rare and possibly life-threatening reaction.

  • Complications from those events led to “fatal outcomes,” according to a company spokesperson.

• Bristol Myers has also voluntarily paused enrollment in autoimmune trials evaluating its own CAR-T treatment zolacabtagene autoleucel, or zola-cel, “out of an abundance of caution,” according to a company spokesperson.

• Both rap-cel and zola-cel are being developed with technology that’s meant to speed up production compared to earlier CAR-T products.

• That technology “could be driving increased cell expansion and the reported toxicities,” wrote William Blair analyst Sami Corwin, though she cautioned in a Monday client note that other factors could be involved too. (BioPharma Dive)

• The company submitted applications to the FDA as well as UK authorities, asking for approval of its Huntington’s disease gene therapy.

• UniQure said it requested priority review, which is typically six months, in its FDA application for AMT-130.

• The submission comes after the FDA walked back its rebuke of the experimental gene therapy under former leadership, allowing UniQure to seek accelerated approval of a potential treatment for the disease, which causes the brain’s nerve cells to break down over time. (Endpoints)

• Ultragenyx had been flying high after its recent FDA approval for Genglycos—but the company has been brought back down to earth with a damaging phase 3 flop.

• The late-stage Aspire trial was assessing its investigational asset apazunersen in Angelman syndrome, a rare genetic condition that affects the nervous system and causes severe physical and learning disabilities.

• The drug missed its primary endpoint of change from baseline in Bayley-4 cognitive raw score, while also falling short in its secondary endpoint, which was net response on the Multidomain Responder Index, a measure of clinical function.

• The flop sent Ultragenyx shares down 43% after hours Wednesday evening on the news. The future of the drug remains in the balance, with Ultragenyx saying that it's now looking over the apazunersen program “in light of this outcome and [will] make a decision on its disposition.” (Fierce Biotech)

• The FDA will add resources to expand oversight at foreign clinical trial sites, aim to improve communication with drug sponsors and strengthen internal reviewer training to help identify clinical trial sites of possible concern, according to the agency’s editorial, which was published in FDA Voices on Wednesday afternoon.

• Notably, the article does not refer to any specific geographical region.

“This is not protectionism, and it is not a geopolitical statement about any particular country’s scientific capacity,” the authors wrote. “Many foreign research institutions produce clinical data of the highest quality, and the FDA actively welcomes and relies on such data.”

“Sponsors should treat the inability to inspect or access all relevant data as a material factor in their regulatory strategy, not an administrative footnote,” the FDA officials wrote.

• Last week, the FDA approved updated vaccines to protect against the predominant strain of the virus.

• Both Moderna and Pfizer received approval for their mRNA vaccines and Sanofi also received approval for a protein-based shot, similar to vaccines that have been around for decades.

"Like the influenza virus, the COVID virus mutates; it changes notably over time, and so we have to update the vaccine in our best attempt to match the strain that we anticipate will be most active during the coming winter," Dr. William Schaffner, a professor of preventive medicine at Vanderbilt University Medical Center in Nashville, said.

• XFG currently accounts for about 40% of cases in the US, according to the latest data from the Centers for Disease Control and Prevention (CDC) in July.

• Moderna's shot is approved for those 6 months through 64 years old with an underlying condition and for all adults aged 65 and older.

• Pfizer's shot is approved for those 5 years through 64 years old with an underlying condition and for all adults over age 65.

• According to the CDC, underlying conditions placing people at risk for higher risk of severe COVID-19 include but are not limited to cancer, asthma, chronic lung disease, diabetes, heart conditions, mental health conditions and pregnancy.

• Public health specialists have said that it is safe to receive a COVID-19 vaccine at the same time as a flu shot or an RSV vaccine, for those who are eligible.