EU regulatory committee recommends 8 new drugs for approval, including Bristol Myers' BCMA CAR-T
At its June meeting, the European Medicine Agency’s Committee for Medicinal Products for Human Use recommended eight new drugs for approval, bringing this year’s count to 50 positive opinions.
While the European Commission doesn’t need to follow the CHMP’s recommendations, it typically does. Here are the drugs that got the committee’s green light this month:
A few months after becoming the first marketed BCMA CAR-T therapy, bluebird and Bristol Myers Squibb’s Abecma (also known as ide-cel) has snagged a CHMP recommendation for conditional marketing approval in relapsed or refractory multiple myeloma. The committee recommends the drug as a fourth-line therapy, after patients have tried an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody.
Abecma’s US approval came about a year after BMS and bluebird got slammed with a surprise refuse-to-file letter. Regulators based their decision on data from the pivotal Phase II KarMMa trial, which showed that among 100 patients taking ide-cel, 72% saw their tumors shrink and 28% achieved a stringent complete response. An estimated 65% of those had remission lasting at least 12 months, the companies said.
“As the first CAR T cell therapy for relapsed and refractory multiple myeloma to receive a positive CHMP opinion, Abecma represents a potential new treatment approach for patients in Europe battling this incurable blood cancer,” said Noah Berkowitz, BMS’ senior VP of cell therapy development, in a statement.
The partners said back in March that the drug would have a US list price of $419,500.
However, rivals like J&J and Legend are catching up. Cilta-cel was granted priority review in May, the same month the partners released Phase II data showing a 95% overall response rate, with a 75% stringent complete response rate. Those data are part of the package J&J is submitting in rolling applications for approvals in Europe and the US.
BMS also came away with another CHMP win, as the committee recommended expanding Opdivo’s label to include the adjuvant treatment of adults with esophageal or gastroesophageal junction cancer following prior therapy.
BioMarin’s vosoritide is still awaiting a decision from the FDA, but the CHMP has recommended an OK for the treatment of achondroplasia, a condition that causes dwarfism.
The CHMP’s recommendation covers patients two years and older until their growth plates are closed, which occurs after puberty when children reach their final adult height.
Upon getting vosoritide across the FDA’s doorstep back in November, BioMarin said the drug spurred annual growth of 1.57 cm over placebo and patients, on average, gained 2.2 cm in height compared to what natural history suggested they would in a Phase III study. Phase II data suggest that the drug helped add up to 9.0 cm in cumulative additional height over 4 and a half years. That was in patients 5 to 18 years old. However, the FDA made it clear that a panel of outside experts had recommended two-year controlled trials across different age groups.
Regulators had initially set an August 2021 PDUFA date, then extended the decision until November after BioMarin submitted two-year results from a Phase III extension study.
Almost a year after the FDA granted MorphoSys and Incyte a quick okay on Monjuvi, the CHMP has given the CD19-targeting monoclonal antibody a recommendation in relapsed or refractory diffuse large B-cell lymphoma (DLBCL).
The conditional marketing authorization would likely come for tafasitamab in combo with lenalidomide, followed by tafasitamab monotherapy in patients who aren’t eligible for autologous stem cell transplant, according to the CHMP’s opinion.
The drug, which would be marketed as Minjuvi in Europe, raked in $22 million from its launch last August to the end of December. Results released last May showed a 59% response rate, a 39% complete response rate, and a median duration of response of 34.6 months — nearly 3 years.
“Patients with relapsed or refractory DLBCL have limited treatment options and often face a poor prognosis. There is an urgent need for effective therapies and if approved, this combination could provide patients in Europe with an important new therapeutic option,” said Malte Peters, MorphoSys’ chief R&D officer.
Incyte and MorphoSys share global development rights to tafasitamab, and Incyte has exclusive commercialization rights to tafasitamab outside the United States.
Belgium’s UCB isn’t expecting an answer from the FDA on its bimekizumab until October. But the CHMP has given it the thumbs-up for the treatment of moderate to severe plaque psoriasis.
Over the last couple of years, UCB has claimed Phase III wins against J&J’s blockbuster Stelara, AbbVie’s Humira, and Novartis’ Cosentyx.
Cosentyx blocks interleukin-17F, one of the IL-17 cytokines involved in psoriasis. The idea behind bimekizumab was that by blocking both interleukin 17s involved in psoriasis — IL-17F and IL-17A — you could have a stronger effect.
UCB says 58.6% of patients who took bimekizumab were completely cleared of skin lesions after 16 weeks, compared to 20.9% of patients on Stelara. It also outperformed Stelara at how many patients were clear after one year and at lesser benchmarks for plaque clearance, with more than 8 out of 10 patients showing 90% improvement, compared to roughly half on Stelara, the company said.
When pitted against a placebo, bimekizumab helped 68% of patients achieve completely clear skin, and more than 90% see a 90% improvement, according to Phase III data released in 2019. For placebo that number was 1.2%.
Back in April, the FDA set a PDUFA date for Oct. 15.
Just as Genentech’s long-running blockbuster Lucentis is set to lose patent protection in Europe, the CHMP has recommended an OK for Samsung Bioepis and Biogen’s biosimilar, dubbed Byooviz.
The committee is recommending the drug for use in a slate of visual impairments including: wet age-related macular degeneration, visual impairment due to diabetic macular edema, proliferative diabetic retinopathy, visual impairment due to macular edema secondary to retinal vein occlusion (branch RVO or central RVO), and visual impairment due to choroidal neovascularisation.
Lucentis was first approved for wet AMD in the US back in 2006. In 2017, it became the first FDA-approved treatment for all forms of diabetic retinopathy, the leading cause of blindness in working-age adults, back in 2017. However, the market’s now crowded with long-acting AMD drugs, including Regeneron’s Eylea and Novartis’s Beovu.
“Biosimilars could help broaden access and offer significant healthcare savings through the treatment of these complex and often debilitating ophthalmic diseases,” said Ian Henshaw, Global Head of Biosimilars at Biogen.
Europe could soon have its first oral inhibitor of hypoxia-inducible factor (HIF) prolyl hydroxylase (PH), according to Astellas and FibroGen.
The CHMP gave roxadustat — which would be marketed as Evrenzo — a positive opinion for the treatment of anemia in patients with chronic kidney disease. The drug is already approved in this indication in Japan, China and Chile, and is under review in the US.
The drug ran into trouble back in April, when FibroGen backtracked on some of its key safety data. According to CEO Enrique Conterno, FibroGen execs became “aware” that their analysis included post hoc changes to stratification factors used to assess the hazard ratio of their drug — essentially manipulating the data to enhance how much the therapy reduced key risks for patients and sharing that false profile with investors and regulators. Using pre-specified stratification factors raised the risk on key safety endpoints, which revolved primarily around MACE, a composite endpoint of all-cause mortality, stroke, and myocardial infarction.
Shares plunged on the news, eviscerating about a billion dollars of FibroGen’s market cap.
Two more generics
The CHMP has also recommended marketing authorization for two generics: Abiraterone Mylan (also known as abiraterone acetate, a generic to Janssen’s Zytiga) for metastatic prostate cancer and Fingolimod Mylan (fingolimod, a generic to Novartis’ Gilenya) for relapsing-remitting multiple sclerosis with high disease activity.