Eight medicines have cleared the final hurdle before a decision on their approval in Europe, having won the backing of the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP).
The Committee recommended granting a conditional marketing authorisation for Novartis’ Zolgensma (onasemnogene abeparvovec) for the treatment of babies and young children with spinal muscular atrophy. The advanced therapy medicinal product (ATMP) is indicated for the rare and often fatal genetic disease that causes muscle weakness and progressive loss of movement.
A positive opinion was also issued for other Novartis products: Atectura Breezhaler (indacaterol/mometasone furoate) and Bemrist Breezhaler (indacaterol/mometasone furoate) for the treatment of asthma; and Seqirus’ Fluad Tetra (influenza vaccine [surface antigen, inactivated, adjuvanted]) for prophylaxis against influenza.
Elsewhere, the CHMP backed Sanofi’s Sarclisa (isatuximab) in combination with pomalidomide and dexamethasone (pom-dex) for the treatment of certain adult patients with relapsed and refractory multiple myeloma. The drug can be used in patients who have received at least two prior therapies including lenalidomide and a proteasome inhibitor and have demonstrated disease progression on the last therapy, after data showed a significant improval in progression-free survival.
Mylan’s biosimilar medicine Nepexto (etanercept) bagged a positive opinion for the treatment of rheumatoid arthritis, juvenile idiopathic arthritis, psoriatic arthritis, axial spondyloarthritis (ankylosing spondylitis, non-radiographic axial spondyloarthritis), plaque psoriasis and paediatric plaque psoriasis.
Finally the CHMP endorsed Celgene’s Zeposia (ozanimod) for the treatment of adult patients with relapsing remitting multiple sclerosis with active disease, and FGK Representative Service's Pretomanid FGK (pretomanid) for the treatment of tuberculosis in combination with bedaquiline and linezolid.

Alnylam has submitted marketing applications for lumasiran on both sides of the Atlantic for the ultra-rare inherited disease primary hyperoxaluria type 1 (PH1).
The firm has completed a rolling submission to the US Food and Drug Administration and marketing authorisation application to the European Medicines Agency (EMA) for use of the RNAi therapeutic.
If ultimately given the green light by regulators, lumasiran would be the first approved therapeutic treatment option for PH1 patients, according to the firm.
The filings include data from the pivotal ILLUMINATE-A Phase III study, in which lumasiran met all primary and secondary endpoints, demonstrating a substantial reduction in urinary oxalate excretion – a key factor in the underlying pathophysiology of PH1 – with an 'encouraging safety and tolerability profile'.
PH1 is characterised by painful and recurrent kidney stones, resulting from excess oxalate production. This excess oxalate can lead to compromised kidney function, resulting in subsequent accumulation of oxalate crystals in bones, eyes, skin and heart, leading to severe illness and death.

Novo Nordisk's Rybelsus (oral semaglutide) has won European approval for type II diabetes, offering adults a new treatment option as the first and only oral glucagon-like-peptide-1 (GLP-1) receptor agonist. The decision allows doctors to prescribe the drug as an adjunct to diet and exercise for adults whose type II diabetes remains uncontrolled. The approval is based on data from 10 PIONEER clinical trials, in which the drug showed “statistically significant reductions in HbA1c vs sitagliptin, empagliflozin and liraglutide and with up to 4.3kg weight reduction,” the firm noted. The most common adverse event observed was mild to moderate nausea, which lessened over time.

EU regulators have accepted for review Arvelle Therapeutics’ marketing application for cenobamate for the adjunctive treatment of focal-onset seizures in adults with epilepsy, based on clinical trials in which the drug cut seizure frequency by 56%. Cenobamate, discovered by SK Biopharmaceuticals and licensed to Arvelle in 2019, is believed to work through a unique, dual, complementary mechanism of action: enhancing inhibitory currents through positive modulation of GABAA receptors, and decreasing excitatory currents by both inhibiting the persistent sodium current and enhancing the inactivated state of voltage-gated sodium channels.

Biogen’s Fampyra (fampridine) has become the first treatment funded by NHS Scotland to improve walking difficulties in adult patients with all types of multiple sclerosis (MS), following a resubmission by the company. The drug was initially turned down by the SMC in late 2018 as it felt there was too much uncertainty surrounding its value for money, but it has now approved NHS funding for its use to improve the walking capabilities of adults with MS with walking disability, but only in circumstances where the approved Patient Access Scheme (PAS) is utilised or where the list/contract price is equivalent or lower than the PAS price.

The EMA’s safety committee (PRAC) has now confirmed that LEO's Picato (ingenol mebutate), a gel for treating the skin condition actinic keratosis, may increase the risk of skin cancer and concluded that its risks outweigh its benefits. The conclusions are based on a review of all available data on the risk of skin cancer in patients using Picato, including results of a study comparing the drug with imiquimod, which showed a higher occurrence of skin cancers, especially squamous cell carcinoma, in areas of skin treated with Picato. The Committee also included that Picato’s effectiveness is not maintained over time. Picato is no longer authorised in the EU.

Menlo Therapeutics has been hit with a setback after a once-daily, oral formulation of serlopitant failed to hit primary goals in two late-stage trials. In both, serlopitant did not induce a statistically significant reduction in pruritus (associated with prurigo nodularis) in patients compared to placebo. “While the data showed a numerical advantage for serlopitant compared with placebo on the primary endpoint, the difference was not statistically significant,” said David Domzalski, Menlo's chief executive. “We will thoroughly analyse these data to better understand the outcome but, at this point, we do not intend to further pursue serlopitant,” he added.

Eli Lilly and Boehringer Ingelheim have received a complete response letter from the US Food and Drug Administration regarding an application to market Jardiance (empagliflozin) as an adjunct to insulin for adult patients with type I diabetes. The decision follows an FDA panel vote last November recommending against the approval of the drug, which called for an additional studies to further back the benefit-risk profile for the SGLT-2 inhibitor. The response letter indicates that the US regulator is unable to approve the companies’ supplemental New Drug Application (sNDA) in its current form. The drug is already on the market for the treatment of type II diabetes.