Shortly after crossing the FDA’s finish line, Ionis Pharmaceuticals’ RNA-targeted hereditary angioedema (HAE) treatment Dawnzera is on its way to breaking ground in the European market with an endorsement from the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP).
Dawnzera, which is under the stewardship of Ionis’ partner Ostuka Pharmaceuticals in Europe and the Asia Pacific region, won CHMP’s favor based on a phase 3 study that showed the drug was associated with an 81% reduction in HAE attacks versus placebo.
The genetic condition is hallmarked by recurrent attacks of severe swelling in various parts of the body and is estimated to affect some 1 in 50,000 people worldwide, according to Ionis.
The CHMP opinion is a “key milestone in advancing access to potentially life-changing treatments for the HAE community,” Otsuka's chief executive in Europe said in a statement.
The drug won its first approval in the U.S. in August amid a recent upswell in new HAE treatments. Along with being the first RNA-targeted treatment for the condition, Dawnzera sets itself apart with its dosing schedule and availability in pre-filled pens for at-home injections.
Now, Otsuka's EU application is off to the European Commission (EC), which will give the final call on approval for those ages 12 and older, CHMP said.
Nods for Eli Lilly, Sanofi and more
Eli Lilly also received good news among CHMP’s group of Nov. 13 decisions, with the Indianapolis pharma scoring an endorsement for its oral selective estrogen receptor degrader (SERD) Inluriyo.
The monotherapy, for patients with locally advanced or metastatic breast cancer with an activating ESR1-mutation, is the second oral SERD to pass muster with regulators in Europe or the U.S. after Italy’s Menarini first broke into the tricky treatment space in 2023.
Lilly’s offering, if approved by the EC, would hold a European label for use in patients who have experienced disease progression after treatment with an endocrine-based regimen, CHMP said.
CHMP’s latest meeting cleared the way for 10 products in total, including two biosimilars and two generics. Others to earn a thumbs-up include Sanofi’s disease-modifying Type 1 diabetes infusion Teizeild, which was classified under CHMP’s priority medicine (PRIME) pathway to speed up drugs that meet an unmet need. The FDA recently took a similar approach with Tzield, giving it an expedited review under the new FDA Commissioner's National Priority Voucher program for a potential label expansion.
Elsewhere, CHMP signed off on a radionuclide generator made by Curium Romania SRL, a whooping cough (pertussis) vaccine made by BioNet Europe and Fondazione Teleth’s Waskyra, the first gene therapy that can treat Wiskott-Aldrich syndrome.
Fondazione Telethon is the first non-profit organization to have spearheaded a drug’s path from research all the way to regulatory approval, it said in a press release.
As for biosimilars, Gan & Lee’s copy of Sanofi’s long-acting insulin Lantus and Theramex Ireland’s version of Amgen’s bone meds Prolia and Xgeva also secured CHMP endorsements, as did Accord’s generic version of Astellas and Pfizer’s prostate cancer drug Xtandi.
Viatris’ generic copy of Takeda’s short bowel syndrome injection Revestive also made the grade at this week's CHMP meeting.
CHMP also announced that it has begun its re-examination of Sanofi’s Rezurock in the third-line treatment of chronic graft-versus-host disease. The company received a negative CHMP opinion on the med last month and quickly pledged to seek a re-review.
Avanex Life Sciences’ bid for its blarcamesine prospect in early Alzheimer’s disease, meanwhile, received a “negative trend vote” by the committee, the company said. Avanex’s shares fell 35% on the Friday news.