DUBLIN – Novartis AG, Bristol Myers Squibb Co. and Sanofi SA were among firms that secured positive opinions from EMA’s Committee for Medicinal Products for Human Use (CHMP) on marketing authorization applications for new therapies, which will progress to formal approvals 67 days from now. Because of the COVID-19 pandemic, the committee’s March meeting was held virtually.
Included in the latest haul is Zolgensma (onasemnogene abeparvovec), the gene therapy developed by Avexis, a subsidiary of Basel, Switzerland-based Novartis, for treating spinal muscular atrophy. Novartis also received positive opinions for two asthma therapies, both based on combinations of indacaterol and mometasone furoate, Atectura Breezhaler and Bemrist Breezhaler. New-York-based BMS got the nod for its oral, sphingosine 1-phosphate (S1P) receptor modulator Zeposia (ozanimod) for treating relapsing-remitting multiple sclerosis (RRMS), having also received FDA approval for the same drug this week. Paris-based Sanofi gained a positive opinion for Sarclisa (isatuximab) in multiple myeloma. A flu vaccine and a new antibiotic for tuberculosis complete the line-up of new products that will shortly enter Europe’s drug and vaccine markets.
The imminent conditional approval of Zolgensma in Europe will arrive about one year after that handed down by the FDA. Both the FDA approval and a recent approval in Japan are limited to patients who under 2 years of age, whereas the CHMP has recommended a broader label, which includes “babies and young children,” without specifying an age limit. The committee’s decision was based on Str1ve-US an open-label phase III trial that enrolled 22 infants less than 6 months of age with non-functional survival motor neuron 1 alleles (SMN1) and with one or two copies of the SMN2 gene, and Start, a phase I trial in patients with two copies of SMN2. Interim data from the SPR1NT trial, an open-label phase III study in 30 presymptomatic infants less than 6 weeks old with multiple SMN2 copies, were also included in the dossier. Another phase III trial, Str1ve-EU, is ongoing.
Although approved centrally, the therapy will be priced on a country-by-country basis, as health care is a national competence within the European Union. Despite the controversy attached to its U.S. price tag of $2.1 million, “nearly all on-label patients” have been approved by their payer for access to the therapy, Avexis said Friday. It also stated that it expects reimbursement to start in Japan by the end of the first half of this year, following approval there earlier this month. Novartis will host a conference call March 30 to discuss its plans for Zolgensma in Europe. A lottery-based system for providing early access to the therapy for 100 patients outside of the U.S. has already drawn the ire of five European health ministers.
The European approval of BMS’s Zeposia extends to active RRMS only, whereas the FDA approval also includes secondary progressive disease, a form of the condition characterized by steady deterioration, which follows the relapsing form. Zeposia is the third S1P receptor modulator to gain approval in MS, following those of Gilenya (fingolimod) and Mayzent (siponimod), both marketed by Novartis. A fourth, ponesimod, developed by the Janssen Pharmaceutical Co. arm of New Brunswick, N.J.-based Johnson & Johnson, was the subject of filings in both the U.S. and Europe earlier this month.
Sanofi’s positive vote for the anti-CD38 antibody Sarclisa follows an FDA approval from earlier this month. The pharma firm is some way behind J&J and Genmab A/S, which have first-in-class anti-CD38 antibody Darzalex (daratumumab), which gained FDA and European approval in November 2015 and May 2016, respectively. It will have its work cut out for it to make meaningful headway in what has become an increasingly crowded market.
Munich-based FGK Representative Service GmbH, acting on behalf of the non-profit TB Alliance, gained a positive opinion for the nitroimidazole antibiotic pretomanid, which will be administered as part of a triple regimen that also contains bedaquiline (Sirturo, J&J) and linezolid (Zyvox, Pfizer Inc.) for treating extensively drug-resistant, treatment-intolerant or non-responsive multidrug-resistant TB infection.
Seqirus, a subsidiary of Melbourne, Australia-based CSL Ltd. picked up a recommendation for Fluad Tetra, an MF59-adjuvanted influenza vaccine that directs an antibody response against the hemagglutinin and neuraminidase antigens of four strains, two A strains and two B strains. It is designed to offer broader protection than existing trivalent vaccines, which protect against two A strains and just one B strain.
Nepexto, a biosimilar version of the tumor necrosis factor alpha (TNF-alpha) inhibitor Enbrel (etanercept) developed by Hatfield, U.K.-based Mylan NV and Mumbai, India-based Lupin Ltd., also gained approval, in multiple inflammatory and autoimmune conditions.