Dive Brief:
- The European Medicines Agency last week recommended nine novel drugs for approval, including Alnylam Pharmaceutical's RNAi therapeutic Onpattro, after the latest meeting of the regulator's Committee for Medicinal Products for Human Use.
- While a positive opinion from the CHMP isn't a final decision, it's a key step toward gaining a marketing authorization from the European Commission. The CHMP also gave a thumbs up to three generic drugs and three biosimilars, in addition to the nine first approvals.
- The news wasn't all positive for drugmakers, though. There were negative recommendations on Dexxience and Eladynos following re-examination, and a restriction on use of Xofigo.
Dive Insight:
Alnylam's Onpattro (patisiran) was one of the drugs securing a recommendation from the EMA last week. The RNAi therapeutic treats hereditary transthyretin-mediated amyloidosis, a rare disease that leads to neuropathy and cardiomyopathy.
Onpattro is pending approval in the U.S., with a Prescription Drug User Fee Act (PDUFA) date of August 11, 2018, and could be the first RNAi drug to hit the market.
Vertex Pharmaceuticals' Symkevi also garnered a recommendation. Symkevi combines tezacaftor with the marketed drug Kalydeco (ivacaftor), and has been approved in the U.S. as Symdeko. This would be the first regimen in the EU to treat the CFTR protein defect in cystic fibrosis patients who have one copy of the F508del mutation and a copy of one of 14 mutations that result in residual CTFR activity.
Bacterial infections are becoming harder to treat as increasing numbers of bacteria become resistant. Tetraphase Pharmaceuticals, though, picked up a recommendation from the CHMP for eravacycline, which is in development for complicated intra-abdominal infections. In the IGNITE 4 study, eravacycline was found to be non-inferior to Merrem (meropenem), one of only a few antibiotics used in this complex infection.
News from the agency wasn't all positive for drugmakers, though. In March 2018, the EMA had given Dexxience (betrixaban) and Eladynos (abaloparatide) negative opinions, and reiterated these on re-examination at the July meeting.
Dexxience, Portola Pharma's drug for the prevention of venous thromboembolism, wasn't recommended due to concerns that the main study failed to show Dexxience's effectiveness when used for preventing blood clots in patients admitted to hospital. There were also worries that patients treated with Dexxience had more episodes of bleeding than those treated with the comparator medicine.
"This was considered an important concern given that the medicine was expected to be used in patients with serious underlying conditions for whom any episode of bleeding could have serious consequences, and Dexxience's long persistence in the body could complicate management of bleeding," the agency said in its outcome of re-examination report.
Radius International's Eladynos also fell short on clinical trial data. According to the EMA's report, the main study didn't satisfactorily show that the drug was is effective at preventing non-vertebral fractures in post-menopausal women. Data from two of the sites was deemed to be unreliable. In addition, the CHMP had concerns about increases in heart rate and palpitations.
"Because most post-menopausal women are at an increased risk of heart problems, the CHMP could not identify a group of patients in whom the benefits would outweigh the risks," the EMA committee said.