VM Biopharma’s Gene Therapy to Treat ALS Approved for Fast Track Development by FDA

VM Biopharma’s Gene Therapy to Treat ALS Approved for Fast Track Development by FDA

The U.S. Food and Drug Administration (FDA) has given Fast Track designation to VM BioPharma’s lead investigational drug candidate, VM202, a potential gene therapy for the treatment of amyotrophic lateral sclerosis (ALS).

VM BioPharm is planning to publish data from its Phase 1/2, open-label trial assessing the safety and tolerability of VM202 (primary endpoints) in people with ALS later this year. The trial’s secondary endpoints included measures of the ALS Functional Rating Scale (ALSFRS-r), an efficacy parameter.

“We are very pleased to announce that the FDA has granted Fast Track designation for VM202 in the potential treatment of ALS. This status underscores the need to expedite potentially new and important treatment options for the ALS community,” said Seungshin Yu, head of new business development of ViroMed Co., VM BioPharma’s parent company, in a press release. “The Fast Track designation, coupled with the recent Orphan Drug designation from the FDA, provides even more momentum in our work to address the urgent and significant unmet medical need that remains in the treatment of people with ALS.”

VM202 is a DNA-based drug intended to improve microvasculature and regenerate nerve cells. When VM202 is injected into patients, it produces a protein called hepatocyte growth factor (HGF), which is to induce angiogenesis — the formation of new blood vessels — and acts as a neurotrophic factor.

The drug is also in clinical testing as a potential treatment for diabetic neuropathy and limb ischemia.

ALS, a disorder that involves the death of neurons, is characterized by stiff muscles, muscle twitching, and gradually increasing muscle weakness that results in difficulty speaking, swallowing, and eventually breathing. The disease’s cause  is not known in 90% to 95% of cases. In others it is an inherited disease, and about half of these genetic cases are due to one of two specific genes.

Fast Track designation is intended to speed the development and review of drugs under study to treat a serious or life-threatening condition and fill an unmet medical need.

 

Margarida graduated with a BS in Health Sciences from the University of Lisbon and a MSc in Biotechnology from Instituto Superior Técnico (IST-UL). She worked as a molecular biologist research associate at a Cambridge UK-based biotech company that discovers and develops therapeutic, fully human monoclonal antibodies.
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Margarida graduated with a BS in Health Sciences from the University of Lisbon and a MSc in Biotechnology from Instituto Superior Técnico (IST-UL). She worked as a molecular biologist research associate at a Cambridge UK-based biotech company that discovers and develops therapeutic, fully human monoclonal antibodies.
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5 comments

  1. Kim Persaud says:

    Please get some treatments approve. Before it’s too late for most people. Why is the Fda taking so long. I need some treatment now, since I have been diagnosed in November 2016.

    • Tim Bossie says:

      Thank you for your comment Kim! We do hope that there is rapid approvals for treatments to help you and others who suffer.

  2. Nina says:

    Dear Doctors,
    Please hurry up, really this destroys people every day. God Bless you in this kind research. I am 30 years old female, also diagnosed 1 year ago and you make me very optimistic, also other clinical trials are under investigation and don’t be afraid friends. I hope it will be treated in the nearest future.

  3. Andreas Deranco says:

    Hope I get VM202 as soon as possible . It’s really painful and VM202
    Are for many people a big hope .
    Regards Andreas

  4. Valerie Evans says:

    My sister has just been diagnosed with ALS. She’s going downhill fast. In February/March of this year she was fine. Now it’s June and her speach is so garbled we can hardly understand her, she has trouble swallowing, her writing is barely legible and she has trouble walking. She has had to quit work and since she is only 63 is not eligible for Medicare and will be losing her work paid health plan soon. New health insurance is going to be very expensive and with a small income she’s going to be in a financially difficult situation. We hope Social Security Disability will kick in soon to help her out. My family can’t lose her. HELP!

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