Mother and Daughter Hugging

Corbus Pharmaceuticals announced today that the European Union has approved orphan designation for lenabasum for the treatment of dermatomyositis. Lenabasum received orphan drug status in the US earlier this year, providing incentives for the continued drug testing necessary for FDA approval.

Like the US, the EU grants orphan designation to drugs that are intended for the treatment of life-threatening or chronically debilitating rare diseases where no satisfactory therapy is currently available.

Lenabasum is a synthetic, oral, non-psychoactive cannabinoid receptor type 2 (CB2) agonist that has shown significant promise for treating dermatomyositis in earlier clinical trials. The drug works by resolving inflammation and fibrosis and halting the production of additional inflammation. One of the values of this treatment is that it does not suppress inflammation like prednisone and other myositis medications, and it has few side effects.

Phase 2 studies in diffuse cutaneous systemic sclerosis (scleroderma), dermatomyositis, and cystic fibrosis suggest that the drug may have clinical benefit. Additional clinical studies are being conducted and/or planned to confirm these preliminary results and support applications for regulatory approval in both the US and in Europe.

See the company’s press release here.

13 comments on “Treatment for dermatomyositis receives orphan designation”

  1. 1
    Jeannette on September 18, 2018

    Prayers being answered? Hopefully, this is a good step in helping people with these issues

  2. 2
    Wanda Spannuth on September 18, 2018

    Is this treatment effective for other types of Myositis?

    1. 3
      Linda Kobert on September 18, 2018

      Wanda, lenabasum is currently being tested just for the skin symptoms of dermatomyositis. We can keep our fingers crossed that once it is approved, there will be other indications for its use, including other forms of myositis.

  3. 4
    Robin sisson on September 18, 2018

    Praise God! Keep the reasearch going!

  4. 5
    Stacy on September 18, 2018

    So it only helps the skin part of dermatomyositis? Does it help with the pain in the muscles or the weakness?? Lord I hope so!! This would answer so many prayers for my daughter!!!

    1. 6
      Linda Kobert on September 19, 2018

      Stacy, the initial trial tested for effectiveness with skin symptoms. It’s our understanding that the larger clinical trial, which hasn’t started recruiting yet, will also include muscle symptoms.

  5. 7
    Ruhangez on September 18, 2018

    All the best in the research.

  6. 8
    Gaius Gracchus on September 21, 2018

    I got off the prednisone, methotrexate and plaquenil by implementing a number of changes including LDN. I had dermatomyositis and decided that wrecking my immune system with the drugs they wanted me on was not a good long term plan, in spite of it working in the short term.
    Glad they are thinking outside the box on this one, too.

    1. 9
      deborah lamartiniere on September 29, 2018

      what is LDN?

    2. 10
      Kay on November 18, 2018

      What is LDN?
      I i’ve been trying to get off prednisone for the last four years I’m on 150 mg every month of Gamunex‘s 16 1/2 mg of prednisone and having a hard time dropping down from there my doctor wants me to start Rituxan infusion in addition to the other one that I’m doing just curious what LDN is .

  7. 11
    Jessica alfaro on September 25, 2018

    Que. Bueno en realidad porque. Dermatomiositis es una enfermedad clasificada como rara y es muy triste y complicada. Dios mediante. Se pinga al alcanze de lis que padecemos de esta enfermedad.

  8. 12
    Linda Kaminski on October 2, 2018

    Yes, please, what is LDN?

    1. 13
      Linda Kobert on October 3, 2018

      LDN = low dose naloxone
      This refers to the off-label, experimental use of the opioid antagonist medication naltrexone at low doses to treat certain conditions. LDN is often used to treat chronic pain.

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