FDA Breakthrough Designation Approval: Challenges & Tips from Roche’s Dr. Niklas Engler

Since being signed into law on 9th July 2012, the FDA’s Breakthrough Designation has changed the face of drug development. In November 2013 Roche’s monoclonal antibody, Gazyva, became the first medicine designated as a Breakthrough Therapy and in the next two years a further 37 approvals were granted. This increasing trend has continued into 2016, with a raft of new drugs given approval in the first two months of the new year.

Roche continue to be at the forefront of the industry and have had a hand in more than one of these new approvals. In January their drug venetoclax (ABT-199), developed in partnership with AbbVie, received a third Breakthrough Therapy Designation for use in combination with hypomethylating agents, to treat patients with first-line acute myeloid leukemia (AML) who cannot receive high-dose chemotherapy. Then just this week it was announced that ocrelizumab (OCREVUS) has been given BTD approval for treatment of patients with primary progressive multiple sclerosis (PPMS).

Early last year we spoke to Dr. Niklas Engler, Head of European Technical Development for Biologics at Roche, about the company’s success with the FDA’s Breakthrough Designation and the challenges it creates.


“The FDA’s Breakthrough Designation has created a direct dialogue between sponsors and the agency. For Gazyva, which attacks targeted cells both directly and together with the body’s immune system, we had weekly exchanges; the speed in which the FDA was working with us was really impressive.”

Technical Support Teams

“The big challenges we face at the moment aren’t necessarily protein production, that is fairly well understood, it’s how technical teams support variations in clinical trials, whether it’s an accelerated programme such as FDA Breakthrough Designation, or heavily gated programmes where you don’t do any technical support for a long time and then have to catch up very quickly.”

Cost and Resources

“Obviously, the financial and personnel investments are huge for such as product so you have to make sure you have the right product in place and all the departments are aligned – technical, clinical and non-clinical development. Because once you jump on that train you accelerate very quickly. If you are not prepared you can really slow down any accelerated advantage you once had.”

Supplying the market

“If you want to go for a large indication like the PD-1 and PDL-1 markets, you need to make sure everything is in place to supply. This is a big topic for the FDA, they don’t want difficulties in supply.”

Want to find out more about this topic? Gargi Maheshwari (Executive Director, Biologics Process Development and Commercialisation, MSD) will be talking on Merck product Keytruda’s FDA Breakthrough Designation at BioProcess International European Summit in Vienna. Her session – “Keytruda – Acceleration of a Breakthrough Therapy…What to do when CMC is on the Critical Path” – is at 9.25am 11th April. Find out more and see the full agenda here.

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