The drug maker said the program would allow for continued reimbursement when patients change insurance plans. A Sangamo-partnered gene therapy for hemophilia A is also in the works.

There are pending requests to add 13 candidates to the tropical diseases that qualify for a priority review voucher, according to a Pink Sheet analysis, as FDA Commissioner Robert Califf faces congressional pressure on the years since the list's last update.

The Swiss pharma company is looking to tap into the experience of ex-BMS leader Giovanni Caforio to help further it US-first commercial strategy.

Deal Snapshot: Ipsen committed up to $1.8bn in upfront and milestone fees to develop novel treatments for rare neurological diseases with Skyhawk’s RNA-targeting small molecule platform.