- The committee responsible for evaluating whether medicines should enter the European market recommended 13 for approval during its September meeting. The greenlighted drugs include Luxturna, an eye disease gene therapy from Spark Therapeutics; Jivi, a hemophilia treatment from Bayer; and galcanezumab, an investigational migraine prophylaxis from Eli Lilly.
- Only one drug didn't pass muster with the committee. Eteplirsen, a treatment for Duchenne muscular dystrophy (DMD) sold in the U.S. under the brand name Exondys 51, received a negative opinion for the second time this year — with the first coming in May. It was under re-examination at the request of its manufacturer, Sarepta Therapeutics.
- Explaining its decision, the committee said there weren't enough clinical data to establish an accurate risk-benefit profile for eteplirsen. As such, "further data were needed to show that the very low amounts of shortened dystrophin produced as a result of Exondys treatment bring lasting benefits relevant to the patient."
DMD is a devastating disease, and one for which there were no approved therapies until eteplirsen came along. Though patient groups applauded Exondys 51's approval, it still ranks as one of the most controversial in recent memory due to the shaky efficacy data that Sarepta submitted in support of the drug.
European regulators, meanwhile, don't look like they're going to follow the Food and Drug Administration's lead. Twice now, the Committee for Medicinal Products for Human Use has advised the European Commission to refuse marketing authorization of eteplirsen. Prospects likely won't get better either, unless Sarepta provides more positive clinical data or works out some kind of agreement with the CHMP.
"Despite the re-examination efforts, the negative opinion was largely expected by [management]," Leerink analyst Joseph Schwartz wrote in a Sept. 21 investor note.
"Therefore, while today's announcement may be perceived negatively, we think it removes an overhang for the remainder of 2018," he added. "Meanwhile, we believe continued discussions with the CHMP could eventually forge a path forward."
Investors may be holding a similar view, as Sarepta's per-share value was down less than 1% at market's open Friday.
Sarepta expects the European Commission to adopt the CHMP's opinion on eteplirsen by year's end. The biotech isn't giving up, however. It intends to seek scientific advice in 2019 as part of a larger plan to bring eteplirsen to the European market.
"We were ... encouraged by the openness of discussion with the SAG and CHMP and their willingness to engage on different approaches to provide additional data to support an eventual approval in Europe," Sarepta CEO Doug Ingram said in a Sept. 21 statement.
Encouraging Sarepta's European pursuits is the success of Exondys 51 in the U.S. Last quarter, the drug raked in $73.5 million. But a broader geographic footprint might be a ways off. Schwartz estimated an eteplirsen launch in the European Union in 2022.