A blockbuster Amgen drug for osteoporosis now carries the FDA's strictest warning that its use can result in dangerously low blood calcium ranges.

This complication, hypocalcemia, is a specific danger for sufferers with superior kidney illness, the Black Field warning mentioned. In these sufferers, this hostile impact resulted in hospitalization, life-threatening occasions, and in some instances loss of life. Extreme hypocalcemia could also be asymptomatic, however signs the FDA warned sufferers and docs ought to look out for embody confusion, irregular coronary heart rhythm, fainting, muscle spasms and weak point.

Prolia, a monoclonal antibody given as a subcutaneous injection each six months, was first permitted in 2010 as a remedy for osteoporosis in postmenopausal ladies at excessive danger of fracture. The drug works by binding to a protein that’s important for the operate and survival of cells concerned within the pure course of by which bone is damaged down and absorbed by the physique. Lowering this bone resorption is meant to extend bone mass and energy.

Whereas the black field warning is new, the chance of hypocalcemia is just not. This complication was a kind of listed on the unique label of the drug. Approval of Prolia was accompanied by a Danger Analysis and Mitigation Technique, an FDA program that imposes strict oversight of a drug to forestall issues of safety. The FDA additionally required Amgen to conduct extra scientific testing to additional assess security.

In 2022, the FDA issued an alert stating that it’s investigating the chance that Prolia may trigger extreme hypocalcemia in sufferers with superior kidney illness requiring dialysis remedy. That warning stemmed from ongoing analysis of Amgen's long-term testing of its osteoporosis drug.

The black field warning comes after an FDA assessment of Facilities for Medicare & Medicaid Companies research discovered that remedy with Prolia led to a big improve within the danger of growing extreme hypocalcemia in contrast with bisphosphonates, an older class of anti-inflammatory medicine. osteoporosis. The FDA mentioned the complication sometimes developed two to 10 weeks after every Prolia injection, with the best danger in weeks two to 5. The company additionally reviewed 25 instances submitted by way of the FDA Opposed Occasion Report System database.

The FDA mentioned sufferers taking Prolia ought to preserve sufficient calcium and vitamin D consumption. For folks with superior kidney illness, the FDA recommends frequent monitoring of blood calcium ranges. The FDA additionally mentioned that physicians ought to take into account Prolia's danger of hypocalcemia within the context of different obtainable osteoporosis medicine.

Final 12 months, Prolia accounted for $2.9 billion in income within the 9 months ended September 30, in keeping with Amgen's most up-to-date monetary report. Amgen additionally sells a more recent osteoporosis drug referred to as Evenity, though hypocalcemia has additionally been reported in sufferers handled with this product. Various osteoporosis therapies embody Eli Lilly's Forteo and Radius Well being's Tymlos. The labels of all these merchandise include black field warnings that flag varied security dangers.

Right here's a abstract of different latest regulatory information:

– Vertex Prescription drugs gene remedy Casgevy obtained FDA approval as a remedy for the uncommon blood illness beta-thalassemia. This approval comes about six weeks after the company first permitted the remedy for the remedy of sickle cell illness. Vertex set a value of $2.2 million for the one-time remedy, the identical value it prices for the sickle cell illness indication. It can compete with Bluebird Bio's Zynteglo, the $2.8 million gene remedy permitted in 2022 to deal with beta-thalassemia.

Authorities in Japan have permitted the AstraZeneca drug danicopan as a brand new remedy for paroxysmal nocturnal hemoglobinuria (PNH), a uncommon blood dysfunction during which the complement system assaults pink blood cells. The oral drug can be marketed below the model identify Voydeya. AstraZeneca already markets the PNH medicine Soliris and Ultomiris, each of which work by blocking a complement system protein referred to as C5. Voydeya targets one other protein referred to as issue D.

Japanese authorities have permitted Voydeya as an add-on remedy for sufferers whose illness is just not adequately managed by a C5 inhibitor. The approval is the primary on this planet for this drug.

—The European Fee has permitted a subcutaneously injected model of Roche most cancers immunotherapy Tecentriq. The drug, which blocks the checkpoint protein PD-L1 on most cancers cells, is offered as an intravenous infusion that takes between 30 and 60 minutes. Roche mentioned the subcutaneous model will take between 4 and eight minutes.

– The FDA has rejected Satsuma Prescription drugs' mixture remedy for migraines, citing manufacturing issues, Satsuma dad or mum firm Shin Nippon Biomedical Laboratories introduced. Satsuma's product candidate, STS101, is a dry powder formulation of the previous migraine drug dihydroergotamine. It’s administered by way of an intranasal gadget meant to offer sufferers with quicker ache aid.

The FDA's full response letter is the newest in a sequence of setbacks for Satsuma. In 2020, STS101 failed a part 3 scientific trial. Evaluation of the research information revealed issues with the gadget and the way in which sufferers used it. Satsuma modified the gadget and performed one other part 3 research.

Manufacturing issues additionally brought about the FDA's rejection of zolbetuximab, a drug from Astellas Pharma developed to deal with adenocarcinoma within the abdomen or gastroesophageal junction. The antibody drug is on the entrance of a aggressive pack of drug candidates designed to focus on claudin 18.2, a protein considerable in abdomen most cancers cells.

Photograph by FDA