- Eight more drugs have received approval recommendations from the committee that advises European regulators on what therapies to clear for market.
- The recommendations include BioMarin Pharmaceutical's Palynziq for a rare metabolic disease, AbbVie's risankizumab for moderate to severe psoriasis and Sanofi's sotagliflozin for adjunct therapy to insulin in certain patients with Type 1 diabetes.
- Three of the eight drugs received positive opinions for conditional marketing authorization. Pfizer will need to gin up additional data for lorlatinib, which recently gained approval in the U.S. as a non-small cell lung cancer (NSCLC) treatment, should the drug ultimately get a thumbs up in the European Union. So will Akcea Therapeutics for its rare disease therapy volanesorsen and Portola Pharmaceuticals' anticoagulant reversal agent andexanet alfa.
The Committee for Medicinal Products for Human Use was good to big drug companies during its last monthly meeting in London before the European Medicines Agency moves to Amsterdam. In addition to recommending approvals for Sanofi, AbbVie, Pfizer and GlaxoSmithKline drugs, the committee also put its support behind label expansions for AstraZeneca's Lynparza (olaparib) and Sanofi and Regeneron Pharmaceuticals' Dupixent (dupilumab).
A positive opinion on risankizumab reinforces AbbVie's high hopes for the drug, which the company sees hitting blockbuster status in the years following its potential approvals in the U.S. and Europe.
Backing risankizumab are four Phase 3 studies that showed significantly more patients achieved PASI 90, a measurement of skin clearance, when taking AbbVie's drug versus placebo or active comparators Stelara (ustekinumab) and Humira (adalimumab).
"The area of, of immune-mediated disease — and psoriasis in particular — is always raising the bar," said Anne Robinson, executive scientific director at AbbVie, in an interview with BioPharma Dive. "In all immune-mediated diseases, it's not going to be good enough to just launch another drug that's going to work as well as what's out there."
If approved, AbbVie intends to market risankizumab in Europe as Skyrizi.
While the psoriasis market holds a medley of treatment options, the opposite is true for people living with a rare disorder known as familial chylomicronaemia syndrome, or FCS.
Akcea is looking to bring a therapy for FCS to market with volanesorsen, which would be branded as Waylivra. The therapy faced a setback in the U.S. last year when the Food and Drug Administration rejected it, in large part due to safety signals seen during a pivotal study.
Paula Soteropoulos, Akcea's CEO, noted that the regulatory process is much different between the FDA and EMA. Importantly, her company was able to submit more data to the CHMP after initially filing volanesorsen.
"It's a very fluid process where we could introduce data later," Soteropoulos told BioPharma Dive. "So we were able to show continued data of patients that were in our open-label extension. That really helped them get comfortable with some of the risk-benefit concerns."
Akcea would need to collect and submit more safety data to European regulators should its drug secure conditional authorization. According to Soteropoulos, an approval could set up volanesorsen for a third quarter launch in the EU, starting in Germany.
The European Commission in July approved another Akcea drug, Tegsedi (inotersen), for the treatment of stage 1 or stage 2 polyneuropathy in adults with hereditary transthyretin amyloidosis. Soteropoulos said that approval gave the company a "base structure" in Europe, which can now work favorably in the potential launch of volanesorsen too.
"All of the things that we put in place for Tegsedi — the supply chain, the patient support — all of those things will be directly applicable to Waylivra," she said.
Soteropoulos estimates that Akcea will hire a few more field sales workers for volanesorsen's initial launch in Germany. Additional workers will likely be hired later on in the European launch, though the total likely will be less than 50, she said.