A narrow miss in a confirmatory trial has threatened to undo Travere Therapeutics’ accelerated approval in the U.S. for Filspari (sparsentan) as a treatment for IgA nephropathy (IgAN).
But that hasn’t stopped the Europe’s Committee for Medicinal Products for Human Use (CHMP) from recommending it for full approval in the condition, which is also known as Berger’s disease.
The CHMP opinion clears the way for Filspari to receive full approval to treat the disease which can cause kidney failure, requiring dialysis or a kidney transplant.
Approved on an accelerated basis by the FDA in February of last year as a first-in-class treatment for IgAN, Filspari came up just short in a confirmatory trial comparing it to irbesartan. While it missed reaching statistical significance on estimated glomerular filtration rate (eGFR), it did reach a range of improvement that is generally considered clinically meaningful, according to analysts at Leerink Partners.
Filspari did however clear the bar on the way the European Medicines Agency (EMA) defines statistical significance, when data from Week 6 to Week 110 are considered, while the FDA standard is Day 1 to Week 110.
After a successful meeting with the FDA at the end of last year, Travere plans this quarter to submit a supplemental new drug application (sNDA) for full approval of Filspari in IgAN..
Travere and CSL, which handles the commercialization of Filspari in Europe, are taking on Calliditas’ Tarpeyo, which became the first drug approved in the indication (on an accelerated basis) in December of 2021 and gained a full FDA nod two months ago, which expanded its label to include all patients with IgAN. The corticosteroid was approved in Europe as Kinpeygo in July of 2022.
Thumbs-up for J&J’s Carvykti
The CHMP also has recommended (PDF) approval of Johnson & Johnson’s Carvykti (cilta-cel) for earlier treatment of relapsed and refractory multiple myeloma (RRMM). The nod would expand its use to patients who have received one prior therapy, including an immunomodulatory agent (IMiD) and a protease inhibitor (PI), have demonstrated disease progression and are refractory to lenolidomide.
Carvykti is the first CAR-T to gain a positive CHMP opinion in the patient population as early as after the first relapse.
“Early resistance to standard treatments is becoming more common in patients with lenalidomide-refractory multiple myeloma, highlighting a need for new options earlier in the course of treatment,” Edmond Chan, M.D., J&J’s regional therapeutic area lead for hematology, said in a statement.
Positive opinion for BeiGene’s Tizveni
Five months after Novartis exited a two-year partnership with BeiGene on Tizveni (tislelizumab), the CHMP has rendered a positive opinion on the PD-1 inhibitor for the treatment of locally advanced or metastatic non-small cell lung cancer (NSCLC) as a monotherapy or in combination with chemotherapy.
Celgene was BeiGene’s original partner for tislelizumab until the company ended its deal in 2019 to help clear the way for its buyout by Bristol Myers Squibb’s for $74 billion because of a conflict of interest with BMS’s Opdivo.
In September of last year, the EU approved tislelizumab for previously treated esophageal squamous cell carcinoma.
Other CHMP nods
The CHMP has given a thumbs-up for Bristol Myers Squibb’s Reblozyl (luspatercept) that would expand its label to include first-line treatment of adults with transfusion-dependent anemia due to low- to intermediate-risk myelodysplastic syndromes (MDS). If approved, it will become the fourth indication for Reblozyl in Europe.
The FDA signed off on Reblozyl in the first-line indication in August of last year. It is approved in both Europe and the U.S. for anemia in adults with beta thalassemia.
CHMP also has recommended Merck’s Keytruda to be approved in combination with chemotherapy as a neoadjuvant treatment, then followed as a monotherapy adjuvant treatment for adults with a high risk of recurrence of resectable non-small cell lung cancer (NSCLC). The FDA approved Keytruda in the indication in October of last year.
An approval would give access to Keytruda to patients in Europe “with earlier stages of non-small cell lung cancer, regardless of PD-L1 expression,” Marjorie Green, Merck’s head of oncology, global clinical development, said in a release.
Also passing muster with the CHMP on Friday was Biogen and Ionis’ Qalsody (tofersen) for patients with amyotrophic lateral sclerosis (ALS) who have a mutation of the superoxide dismutase 1 (SOD1) gene. The FDA blessed the treatment in April of last year.
A month after Japan became the first country to approve AstraZeneca’s Voydeya (danicopan), meanwhile, the CHMP has recommended its authorization in Europe as the first oral treatment against residual hemolytic anemia in patients with the rare genetic disorder paroxysmal nocturnal hemoglobinuria (PNH). Voydeya is part of the EMA’s priority medicines (PRIME) program, which allows early regulatory support for promising medicines that can address unmet needs.
Also clearing the CHMP bar was Incyte’s Zynyz (retifanlimab) to treat Merkel cell carcinoma, a life-threatening type of skin cancer.
Two CSL Seqirus’ vaccines were endorsed as well to combat avian flu—Celldemic (zoonotic) and Incellipan, a pandemic preparedness shot.
Each of the recommendations now go to the European Commission for a final signoff.