After a rejection by the FDA in June, it looks like Orphazyme A/S is headed for disappointment in Europe too with arimoclomol for Niemann-Pick disease type C, a rare and potentially fatal inherited condition in which fat builds in tissues and organs. The Copenhagen-based company said it was summoned before experts to give an “oral explanation” about the drug, something that only occurs if the European Medicines Agency’s CHMP has developed major doubts during its review.
A sense of normalcy is returning to the U.K., at least from a regulatory perspective. The U.K.’s Medicines and Healthcare Products Regulatory Agency will continue to support COVID-19 clinical trials with ongoing regulatory and scientific input, but all further applications and meeting requests for COVID-19 products will be considered according to usual timelines rather than on an emergency basis, the agency said Feb. 23.
Paving the way for wider dissemination of up-to-date information on drugs approved in the EU, the European Medicines Regulatory Network adopted a common standard for the electronic product information (EPI), which includes the package leaflet for patients and the summary of product characteristics for health care professionals.
LONDON – Two years into the pandemic and the number of new drugs approved by the EMA fell from 97 approvals in 2020 to 92 in 2021. But both years are still well up on pre-COVID-19 times in 2019 when 66 products got the nod, according to the EMA’s annual human medicines report.
In the latest chapter in an ongoing contract dispute between AOP Orphan Pharmaceuticals GmbH and Pharmaessentia Corp., the German Federal Court of Justice this week set aside the €143 million (US$162.8 million) in damages awarded to AOP, citing procedural flaws in quantifying the product supply and damages.
In one of its familiar U-turns, the U.K. National Institute for Health and Care Excellence (NICE) has recommended NHS England should fund a rare disease gene therapy from Orchard Therapeutics plc, considered to be the world’s most expensive drug. The list price for Libmeldy (atidarsagene autotemcel) in England and Wales is £2,875,000 (US$3.9 million), making it the most expensive drug that NICE has ever evaluated.
LONDON – The first human challenge study of SARS-CoV-2 infection has reported initial results, showing it is safe to infect healthy volunteers with the virus in controlled conditions, and paving the way for the model to be used to accelerate clinical trials of COVID-19 vaccines, antivirals and diagnostics.
Blueprint Medicines Corp.’s cancer drug Ayvakyt (avapritinib) looks set to gain an expanded label in Europe, amid a flurry of decisions from the European Medicines Agency’s CHMP scientific committee. Late last week the CHMP gave a positive opinion for Ayvakyt for treatment of adults with advanced systemic mastocytosis, meaning the drug is likely to gain a further European indication in the coming weeks.
LONDON – After six years in development, EMA’s new clinical trials information system (CTIS) was switched Jan. 31, bringing to life a law enacted in 2014 to create a single, harmonized regulatory system across the EU. As the embodiment of the Clinical Trial Regulation, CTIS will underpin a long-awaited simplification of the process for approving trials. Rather than separate applications to different national regulators, a single application delivered via CTIS can lead to simultaneous regulatory and ethics approvals in up to 30 countries.
Bristol Myers Squibb Co.’s Breyanzi (lisocabtagene maraleucel) CAR T-cell therapy has passed muster with European regulators as a therapy for relapsed or refractory large B-cell lymphoma, setting up a likely European marketing authorization in the coming weeks.