First ever Breakthrough Therapy drug is approved by FDA

In Clinical Development, Regulation & Government by Tim Peplow

breakthrough therapy gazyva (flickr net_efekt)

The first FDA-designated breakthrough therapy has been approved by US regulators, it was announced last week. Roche’s Gazyva (obinutuzumab) coupled with chlorambucil was given the green light for the treatment of blood cancer chronic lymphocytic leukaemia, and industry commentators expect the orphan drug to be a worthy successor to blockbuster drug Rituxan.

With Rituxan’s patent expiry looming, it had been important for Roche to score a new potential blockbuster to take over the reigns. With Gazyva, Roche may well have one on their hands, as analysts have predicted peak sales between $1.5bn and $2.5bn.

Since Gazyva is the first drug with breakthrough therapy designation to receive FDA approval, there has been much interest around the impact that the BTD had on accelerating the drug’s approval. “This approval reflects the promise of the Breakthrough Therapy Designation program, allowing us to work collaboratively with companies to expedite the development, review and availability of important new drugs,” said Richard Pazdur, FDA. On top of the breakthrough therapy designation, Gazyva had also been granted priority review and orphan drug status.

The approval was based on a study of 356 participants that showed Gazyva demonstrated significant improvement in progression free survival over chlorambucil alone – 23 months versus 11.1 months.

Read the press release from Roche >