It was announced February 17, 2016, that the drug, ocrelizumab made by Roche and Genentech pharmaceutical companies, has been given Breakthrough Therapy Designation for the treatment of primary progressive MS by the Food and Drug Administration (FDA). Ocrelizumab studies Opera and Oratorio had positive results in treating a certain population of people with MS who were fairly early in their progression state and also in relapsing forms of MS. Ocrelizumab is the first investigation medicine to receive Breakthrough Therapy Designation, according to information from Genentech.
This news created much excitement back in Fall 2015 at a world gathering of MS specialists, and I had the opportunity to speak with Dr. Peter Chin, Principal Medical Director for Genentech-Roche Global Neuroscience Development, about this drug development and you can find much more details about the studies and drug in Interview on the PPMS Oratorio Study as well as another great explanation by StephanieBuxhoeveden, in Ocrelizumab Shows Promise for PPMS. Since that time it has been quiet, but it is anticipated the company will file for approval to sell ocrelizumab under the name of Ocrevus by mid-2016 since it has been granted this special status.
The following comes from the FDA website and explains the Breakthrough Therapy Designation:
“On July 9, 2012 the Food and Drug Administration Safety and Innovation Act (FDASIA) was signed. FDASIA Section 902 provides for a new designation – Breakthrough Therapy Designation. A breakthrough therapy is a drug:
- intended alone or in combination with one or more other drugs to treat a serious or life threatening disease or condition and
- preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development.
If a drug is designated as breakthrough therapy, FDA will expedite the development and review of such drug. All requests for breakthrough therapy designation will be reviewed within 60 days of receipt, and FDA will either grant or deny the request. ”
Coincidentally, this announcement comes the same week as specialists from around the world gathered at the Americas Committee for Treatment and Research in Multiple Sclerosis (ACTRIMS) forum, convened to discuss progressive forms of MS. Their topic for research discussions is Progressive MS: From Bench to Bedside and Back. This announcement about ocrelizumab will most certainly add to their discussions. According to Genentech “New data from the Phase III study in PPMS (ORATORIO) will be presented at the ACTRIMS Forum on Friday, February 19th.“
Although this is great news for many people living with primary progressive MS, not all will benefit from its use – from the clinical trials data, it appears the drug was successful in treating people who had lower EDSS scores and we will have to wait a bit longer to learn about recommended uses of Ocrevus. It is nice to think though there will be an FDA approved disease modifying therapy option for PPMS – it’s about time!
Wishing you well,