Fast Track and Orphan Drug Status Granted to Elezanumab Phase 2 Trial for Spinal Cord Injury

Text marked in blue are links to related websites where I found the information I am discussing. Feel free to click, the link will be brought up in a new tab so you won’t loose your place while reading.

I heaved a sigh of relief Monday and I posted and shared my blog entry, that I posted two days behind the schedule I set myself. Spasticity was bad Sunday, I didn’t do the best job with my preschool lesson for my son, but I had at least gotten my blog posted, and shared. My parents took the kids on a walk as I took my herbal remedies and relaxed.

Then a member of the group I established on Facebook, Remyelination- Reaching for the Possible in Multiple Sclerosis posted this article. AbbVie Receives Orphan Drug and Fast Track Designations from the U.S. Food and Drug Administration for Elezanumab, an Investigational Monoclonal Antibody RGMa Inhibitor, for the Treatment of Spinal Cord Injury.

If you are unfamiliar with elezanumab and the phase 2a clinical trial I am taking part in please visit my previous blog post: Going Forward: My Participation in the Clinical Trial for the Remylenation Medication Elezanumab

I quickly read through the article and tried to make sense of it. Member’s questions were already coming in. What does this mean? Will we have access to elezanumab sooner? Will this fast track of the trial make the medication available sooner that the Multiple Sclerosis trials for Elezanumab? My thoughts started racing as my overtired brain tried to piece together the terms I was unfamiliar. What is Orphan Drug Status, and what exactly does fast track mean. Members of the group asked if this would get us access to the drug sooner, as an off label use medication to treat our multiple sclerosis?

The terms that I didn’t yet understand from the article where namely, orphan drug stats and fast track status.

Orphan Drug status can be granted by The FDA to drugs that are used to treat a patient population of less than 200,000 people. It qualifies the maker of the drug for government assistance and tax breaks because given that it is for a rare disease or disorder, there may not be enough market incentives (not enough funding) to finance the trial.

Fast Track status must also be applied for by a drug maker. The FDA grants Fast Track status to drugs that it deems to meet an unmet medical need, demonstrate superior effectiveness to treatments already available, or other criteria listed on their website. A drug that is granted fast track status is eligible to some perks that other trials just have to wait for The FDA to bet back to them.

-More frequent meetings with FDA to discuss the drug’s development plan and ensure collection of appropriate data needed to support drug approval.

-More frequent written communication from FDA about such things as the design of the proposed clinical trials and use of biomarkers

-Eligibility for Accelerated Approval and Priority Review, if relevant criteria are met

Rolling Review, which means that a drug company can submit completed sections of its Biologic License Application (BLA) or New Drug Application (NDA) for review by FDA, rather than waiting until every section of the NDA is completed before the entire application can be reviewed. BLA or NDA review usually does not begin until the drug company has submitted the entire application to the FDA

fda.gov

Ocrevus was granted fast track status by The FDA on Jan 30, 2016. Ocrevus most likely qualified because it was a drug that also met an underserved need, being the first B cell Targeting drug to treat multiple sclerosis. It was also the first drug to show efficacy in slowing down disease progression in people with PPMS. The FDA approved Ocrevus for the treatment of RRMS and PPMS on March 28, 2017. It took over a year to go from receiving fast track status until its approval for the treatment of MS. However, if fast track status had not been given perhaps the wait for the drug would have been longer.

I know many become excited at seeing this new information about elezanumab, even though it is for the spinal cord injury trial. I got incredibly excited too as I look forward to the day that it is elezanumab is available outside of the trial setting. It is incredibly hard to wait. I see people in the group doubting if they can keep going for years while waiting for the remaining phase three trial and FDA approval.

I am waiting with great anticipation the day when I can know for sure that I will be included in the phase 3 trial of elezanumab. Until then I look forward to the results getting published at the completion of the trial and knowing that yes, it really was this risk and gamble that I took with my life. I weighed the costs of the uncertainties of a trial of a new drug, with the potential gain. I think I chose right. Even if I did receive the placebo, I have not lost anything in contributing to the research. I have gained experience and I’ve gained a community of people looking forward to when remyelination will both be a word recognized by spell check and a reality for people living with multiple sclerosis.

If you are interested in joining the conversation surrounding remyelination, please join my Facebook Group, Remyelination- Reaching for the Possible in Multiple Sclerosis. Thank you for my fellow group members, for sharing this advancement in the research of elezanumab and thank you for helping me continue the conversation!

Stay safe, and may God be with you till we meet again!

My Tandem Walk has markedly improvements since being involved in the Elezanumab phase 2 trial. Previously to this trial, I failed the tandem walk for 15 years at my neurologist appointments.

2 thoughts on “Fast Track and Orphan Drug Status Granted to Elezanumab Phase 2 Trial for Spinal Cord Injury

    1. This article is particularly informative. It discusses why our bodies fail to remyelinate. Not only does MS attack our myelin sheath but it plays an active role in preventing our body from remyelination. However, the article discusses the drugs in clinical trial phases, working at stopping this blocking effect to remyelination that MS is producing in our bodies. These ideas are complex, and are hard to digest, especially with cog fog. I plan to write a blog post, posting hopefully by Saturday.

      Here is what the article says specific to elezanumab.

      “Elezanumab is another humanized monoclonal antibody currently tested in phase 2 clinical trials for RMS (NCT03737851) and PMS (NCT03737812). This antibody is directed against the membrane-bound repulsive guidance molecule A (RGMa), which promotes inflammation and inhibits CNS regeneration and remyelination mainly via the multifunctional target receptor neogenin [38]. Anti-RGMa treatment leads to a decreased T cell proliferation, a decrease in pro-inflammatory interleukin production, a functional recovery in experimental autoimmune encephalomyelitis (EAE) [39] and to a prolonged conversion time to the secondary progressive disease phase [40].
      Hope this helps! 🧡😊

      Like

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