good news on the horizon about ocrelizumab and (even rituxan) for progressive MS.

[Lazarus]

National Multiple Sclerosis Society
News
Ocrelizumab Granted “Breakthrough Therapy Designation” for Primary-Progressive MS by FDA. UPDATE: Priority Review Granted by Dec. 28, 2016
June 28, 2016

UPDATE: FDA has granted Priority Review Designation, with a decision target of December 28, 2016.

Originally released February 17, 2016

Genentech, a member of the Roche Group, has announced that the experimental therapy ocrelizumab has been granted “Breakthrough Therapy designation” by the U.S. Food and Drug Administration (FDA) for the treatment of people with primary-progressive MS. This designation means that once Genentech files for approval of ocrelizumab to treat primary-progressive MS, the review process can be expedited. According to a February 16 press release, Genentech plans to pursue marketing approval for both primary-progressive MS and relapsing multiple sclerosis.

“We are encouraged by this news and look forward to following the FDA’s actions regarding ocrelizumab,” says Timothy Coetzee, PhD, Chief Advocacy, Services and Research Officer at the National MS Society. “People with progressive MS need solutions and we are hopeful that this is the first of many treatments that will be developed for this form of MS.”

Breakthrough Therapy designation can expedite the development and review of therapies intended to treat a serious condition when clinical evidence indicates that the treatment may demonstrate substantial improvement over available therapy. The designation was granted to ocrelizumab based on phase III trial results presented at the European Committee for Treatment and Research in MS in 2015. Compared to placebo, ocrelizumab significantly reduced the risk of progression of clinical disability by 24% in 732 people with primary-progressive MS. Read more about these results.

Ocrelizumab is a monoclonal antibody that binds to a molecule (CD20) on the surface of immune cells called B cells, and depletes them from circulation. B cells have several functions including making antibodies, and they may play a role in immune-system mediated damage to brain and spinal cord tissues in MS. Ocrelizumab is administered by intravenous infusion every 6 months. There currently are no FDA-approved disease-modifying therapies to treat primary progressive MS, which is characterized by steady worsening of neurologic functioning, without any distinct relapses or attacks, or periods of remission.

Frequently Asked Questions Related to “Breakthrough Therapy” Designation of Ocrelizumab to Treat Primary-Progressive MS

Q: What is Breakthrough Therapy designation?
A: The Food and Drug Administration has developed four approaches to making treatments available as rapidly as possible when treatments are the first available treatment or if the therapy has advantages over existing treatments. Read more about these on the FDA website. Breakthrough Therapy designation can expedite the development and review of therapies intended to treat a serious condition when clinical evidence indicates that the treatment may demonstrate substantial improvement over available therapy. This designation makes the treatment eligible for all Fast Track designations, intensive guidance on an efficient drug development program, and organizational commitment involving senior managers. It does not make the treatment automatically eligible for priority review, which means FDA’s goal is to take action on an application within 6 months (compared to 10 months under standard review).

Q: Does this mean that ocrelizumab is approved to treat primary-progressive MS?
A: No, this means that once Genentech files with the FDA for approval of ocrelizumab to treat primary-progressive MS, the review process can be expedited. According to a February 16 press release, Genentech plans to pursue marketing approval for both primary-progressive MS and relapsing multiple sclerosis, and will submit data from three phase III studies to the FDA in the first half of 2016.

Q: Will ocrelizumab be approved to treat secondary-progressive MS?
A: At this point, Genentech plans to pursue marketing approval for both primary-progressive MS and relapsing multiple sclerosis. Relapsing MS includes people with secondary-progressive MS who are experiencing relapses. Ocrelizumab reduced relapse rates by 46% to 47% after two years compared to the established MS treatment, Rebif® (interferon beta-1a, EMD Serono) in two phase III studies in people with relapsing MS. Read more about these results. Ocrelizumab has not been studied so far in people with secondary-progressive MS who are not experiencing relapses. Breakthrough designation only applies to primary-progressive MS.

Q: When will ocrelizumab become available?
A: It is not possible to say if or when ocrelizumab may become available. The company has stated plans for applying for regulatory approval of ocrelizumab in the first half of 2016. Even with an expedited review, the timeline for successful marketing approval depends on many variables, such as whether the application is accepted by the FDA or whether the agency requests additional information before the application is deemed complete. Then the FDA needs time to evaluate the results and make a decision about whether the therapy can be marketed.

Q: What role, if any, did the National MS Society play in the development of ocrelizumab?
A: The Society did not play a specific role in the development of ocrelizumab. However, the Society has funded early studies that increased understanding of the role of B cells in the immune attack of MS, as well as an early clinical trial of rituximab, a therapy similar to ocrelizumab. The Society continues to fund researchers exploring the importance of B cells, in relapsing and progressive MS. The Society also provides leadership to the International Progressive MS Alliance, which is funding a study testing rituximab in progressive MS.

[SuzE-Q]

Thanks, Linda.

I wonder if this is what Peg's considering?

[Lazarus]

Thanks, Linda.

I wonder if this is what Peg's considering?

Hi,
I am not sure. I have trouble following the chit chat threads which must be where Peg's information is. I will try to look for it.

[agate]

I think it might be siponimod?

[Lazarus]

Yes Agate, the name is siponimod..First I have heard of it.


Basel, August 25, 2016 - Novartis today announced the Phase III EXPAND study, evaluating the efficacy and safety of oral, once-daily, BAF312 (siponimod) in secondary progressive multiple sclerosis (SPMS), met its primary endpoint of a reduction in the risk of disability progression, compared with placebo. The EXPAND study represents the largest randomized, controlled study in SPMS to date.[1]

"SPMS is a particularly disabling form of MS, and there is a need for effective treatment options to help delay disability progression in those living with the condition," said Vasant Narasimhan, Global Head of Drug Development and Chief Medical Officer for Novartis. "The positive EXPAND data are encouraging for a disease with such a high unmet need. We look forward to sharing the results at the upcoming ECTRIMS congress, and thank all of the study participants and investigators."

Topline results of the EXPAND study, including primary and key secondary endpoints, will be presented as a late breaking oral abstract at the 32nd Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS), September 17th, in London, UK. Novartis will complete full analyses of the data and evaluate next steps in consultation with health authorities.

About the EXPAND study
The EXPAND study is a randomized, double-blinded, placebo-controlled Phase III study, comparing the efficacy and safety of BAF312 versus placebo in people with secondary progressive multiple sclerosis (SPMS).[2] The EXPAND study is the largest randomized, controlled study in SPMS to date.[1] The study included 1,651 people with SPMS from 31 countries. Patients were randomized to receive either 2mg BAF312 or placebo in a 2:1 ratio respectively.[2]

The primary endpoint of the study was an improvement in the time to three-month confirmed disability progression, as measured by the expanded disability status scale (EDSS), versus placebo.[2] Secondary endpoints included delay in the time to six-month confirmed disability progression versus placebo, the time to confirmed worsening of at least 20% from baseline in the timed 25-foot walk test (T25FW), T2 lesion volume, annualized relapse rate (ARR), and the safety and tolerability of BAF312 in people with SPMS.[2]

About BAF312 (siponimod)
BAF312 (siponimod) is a selective modulator of specific types of the sphingosine-1-phosphate (S1P) receptor.[3] The S1P receptor is commonly found on the surface of specific cells residing in the central nervous system (CNS), that are responsible for causing CNS damage that drives loss of function in secondary progressive MS (SPMS).[3] BAF312 enters the brain and by binding to these specific receptors, may prevent the activation of these harmful cells, helping to reduce loss of physical and cognitive function associated with SPMS.[3]-[6]

[agate]

I'm hopeful about siponimod. I always felt that those other drugs failed to take account of those who have MS but no longer have relapses, and as it turned out they really weren't meant for SPMS anyway. Siponimod seems to be specifically aimed at SPMS.

[Howie]

I hate to be the cold water on the hopeful new drugs, but here goes.

I think the cure was found long ago. MS is a moneymaker for the drug companies, why would they want to stop it? But develop new drugs that don't quite cure, just make things better, one month at a time, for a big price.

Let me be wrong, but money drives EVERYTHING in this world. Why would MS be any different?

[agate]

But cures have been found for a number of severe diseases. The polio vaccine has almost wiped polio off the face of the earth, and there has been very effective medicine against TB, for instance.At least some disease cures and vaccines must get past the barriers of a money-dominated world.

[Pegakafarmgirl]

Thanks, Linda.

I wonder if this is what Peg's considering?

yup,, that's the drug, I am going to try,, hopefully it gets here sooner then later

[agate]

You'll be getting ocrelizumab or Ocrevus? There's been a lot of excitement about this drug. They keep improving these drugs but the pace is SO slow.

[Sunshine]

Howie, I think conspiracy theorists also say that about cancer.

I don't believe it for a minute.

There are honest hard working scientists at pharmas. Someone would've blown the whistle by now if the pharmas had the cure but suppressed it.

[Howie]

BBS, I hope you are right.

[Virginia]

I just finished reading a book by Ann Romney "In It Together". Our StillstAnning had talked about it on here. Ann Romney is involved along with Montel Williams and Meredith Viera (whose husband has MS) in research alongside some leading Scientist and Neurologist. Of course there are others on the board also, but I can remember at least these 3 that have every reason to want a cure. So, I do feel that there are people out there trying.

[stillstANNding]

I haven't read the book or mentioned it. Maybe Bbs? I did mention the new MS Center.

And there is no cure for polio but a very good vaccine to prevent it.

ANN

[Virginia]

Sorry Ann, I thought you had also read the book. I thought it was you that mentioned it giving some insight into her relationship with Mitt, though not at all specifically about that. It was mainly her journey. I enjoyed the book. It did talk about the center and the Doctor who has been her MS specialist. I think his name is Dr. Weiner. Have you had the opportunity to see him? He works there and is part of the team (as I understand it) along with some scientist who are now doing the research. I think Dr. Weiner still sees patients. She thinks he is great.