Pentostatin

The once-daily tablet hinders proteins that trigger the severe inflammatory response linked to chronic GVHD. Credit: fizkes / Shutterstock.com. The National Health Service (NHS) in the UK has expedited the availability of the new drug, Belumosudil (Rezurock), for cancer patients with chronic graft versus host disease (GVHD). The decision provides a treatment that can be administered in the at-home setting, reducing hospital visits. Administered as a once-daily tablet, Belumosudil hinders proteins that trigger the severe inflammatory response linked to chronic GVHD. Clinical trials have demonstrated that up to three-quarters of the subjects responded to the therapy, with significant effectiveness in treating symptoms affecting the mouth, intestine or joints. The treatment is accessible through the NHS England’s Innovative Medicines Fund, which fast-tracks the delivery of the most promising non-cancer drugs to patients. See Also: AbbVie licenses OSE’s chronic inflammation therapy for $48m upfront DNTH-103 by Dianthus Therapeutics for Chronic Inflammatory Demyelinating Polyneuropathy (CIDP): Likelihood of Approval Chronic GVHD is a rare but serious condition that can occur after a life-saving stem cell transplant. White blood cells in the donated tissue attack the patient’s organs and weaken their immune system. Approximately one-third of patients who receive stem cell or bone marrow transplants for blood cancers such as leukaemia and lymphoma can develop chronic GVHD. Around 1,200 patients in England undergo such transplants each year. Around 400 go on to develop the condition, which is sometimes mild but for one in ten cases is fatal. For half of patients with chronic GVHD, existing therapies such as pentostatin, rituximab and imatinib are ineffective. Belumosudil will now be made available for 200 severely affected patients who have not responded to two previous therapies. This development comes after the NHS decided to fast-track the funding for the drug based on clinical guidance from the UK National Institute of Health and Care Excellence ( Nice ). Belumosudil is the fourth therapy offered to patients through the Innovative Medicines Fund. In September 2023, the NHS used the fund to roll out Sebelipase alfa, a therapy for treating Wolman’s disease, a rare genetic ailment. NHS England specialised services medical director James Palmer stated: “This rollout is a real breakthrough for patients with graft versus host disease and it will help people living with this debilitating condition effectively manage their symptoms and enhance their quality of life and daily functioning. “It’s great news that the NHS has been able to fast-track funding for a treatment which will have a significant impact for hundreds of patients over the coming years.”

The US Centers for Medicare and Medicaid Services (CMS) has quietly updated the list of Part B prescription medicines subject to penalties because their prices rose faster than the rate of inflation. In an update late last week, the agency cut seven medicines from its original list of 27 that was unveiled on March 15.A provision in the Inflation Reduction Act (IRA), which came into force last year, penalises pharmaceutical companies for charging prices that go up faster than inflation for people with disabilities or those 65 and older who are on the government's Medicare health programme (for more, see ViewPoints: Inflation Reduction Act's myriad impacts to reverberate across pharma). The move is expected to lower out-of-pocket costs for some Medicare recipients by between $1 and $372 per average dose, as of April 1, depending on their individual coverage, according to an agency press release updated on March 30.Gilead cell therapies knocked off The seven drugs that have been cut from the original list include Gilead Sciences' CAR-T therapies Yescarta (axicabtagene ciloleucel) and Tecartus (brexucabtagene autoleucel); Stemline Therapeutics' Elzonris (tagraxofusp-erzs) used for blastic plasmacytoid dendritic cell neoplasm; Shionogi's Fetroja (cefiderocol), an antibiotic to treat complicated urinary tract infections; Arcotech Biopharma's Folotyn (pralatrexate) for relapsed or refractory peripheral T-cell lymphoma; Kamada's WinRho SDF (Rhₒ(D) immune globulin) to boost platelet counts in order to prevent excessive haemorrhage; and Bausch + Lomb's Xipere (triamcinolone acetonide), which is prescribed for macular oedema associated with uveitis.CMS did not give a reason for why the original list was scaled back. Other products that remain include all five Pfizer drugs that were on the original list, namely Atgam (antithymocyte globulin equine), Bicillin L-A (penicillin G benzathine), Bicillin C-R (penicillin G benzathine and penicillin G procaine), Fragmin (dalteparin) and Nipent (pentostatin), as well as Johnson & Johnson's Rybrevant (amivantamab). Companies whose products have been flagged will be required to pay Medicare the difference in the form of a rebate ranging from roughly 14% to 20%. Meanwhile, CMS has said that by September 1, it plans to publish the first 10 Medicare Part D drugs that will be subject to government price negotiations starting in 2026.