• Along with rolling out a $1.8 million price tag in Europe, Bluebird Bio said last Friday its newly approved gene therapy would launch in early 2020, later than initial expectations from Wall Street analysts.
• On the Friday call, Bluebird’s chief commercial officer Alison Finger said the company is working with European regulators to “finalize and narrow our drug product specification and our commercial drug manufacturing process.” She said the company agreed to tighten the process before treating commercial patients as a post-approval commitment with the European Medicines Agency.
• SVB Leerink analyst Mani Foroohar lowered his Bluebird share price target about 15% on Tuesday, citing the commercial launch delay as a primary reason. “The unanticipated delay due to required changes to manufacturing processes introduces new uncertainty into the Zynteglo launch and execution across the broader [Bluebird] platform,” he wrote in a note to clients.

Manufacturing challenges have been a key concern with in the gene therapy space. Earlier this month, the head of the FDA’s Center for Biologics Evaluation and Research called manufacturing his top concern for gene therapies moving forward.

CEO Nick Leschly diminished the delay, saying 2019 will lay the groundwork for 2020, when things will get more interesting. Bluebird’s revised timeline is to enroll the first patients by year’s end and treat the first commercial patients in early 2020 with Zynteglo.

The EMA conditionally approved the gene therapy on June 3 for people with transfusion-dependent beta-thalassemia, a rare, inherited blood disorder. While a key focus of Friday’s call was the $1.8 million pricing plan, spread over five years based on continued effectiveness at 315,000 euros per year, manufacturing concerns crept in as well.

In a statement to BioPharma Dive, the biotech reaffirmed what was said on the call, calling such post-approval requirements common for complex drugs like Zynteglo.

“We plan to finalize these changes with EMA in parallel with our ongoing launch and market access activities in the various European markets,” a spokesperson said in an emailed statement, “with a goal of enrolling our first patients at one of our [Qualified Treatment Centers] by the end of 2019 and treating commercial patients in early 2020.”

Raymond James analyst Dane Leone also saw the launch delay as an explanation for Friday’s sell-off in Bluebird’s stock, which dropped 5%.

“The delay itself is relatively minor in the scheme of the full trajectory of the drug program expected over the next several years, but any opening for competitive efforts to close the distance on Zynteglo will elicit concern,” Leone wrote in a June 14 note.

Even with the regulatory OK in Europe, Bluebird appears poised to remain a pre-commercial biotech for the rest of 2019.