Pamrevlumab, Potential Treatment for Fibrosis in DMD Patients, Named Orphan Drug by FDA

Pamrevlumab, Potential Treatment for Fibrosis in DMD Patients, Named Orphan Drug by FDA

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to pamrevlumab (FG-3019), FibroGen‘s potential anti-fibrosis treatment for Duchenne muscular dystrophy (DMD) patients.

Pamrevlumab is a human monoclonal antibody that blocks the activity of the connective tissue growth factor (CTGF), a protein involved in tissue scarring (fibrosis) linked to the development of several disorders, including idiopathic pulmonary fibrosis (IPF), pancreatic cancer and DMD. It is known as an anti-CTGF antibody .

DMD, the most common type of muscular dystrophy, is a genetic disease that leads to progressive deterioration of muscle fibers. Most patients become non-ambulatory (unable to walk by themselves) between the ages of 6 and 13. At this stage, they tend to develop scoliosis and joint contractures (limited joint mobility), and become more susceptible to heart disease and respiratory failure.

Most disease-modifying DMD medications now available or under testing are meant for patient  still able to walk independently (ambulatory); pamrevlumab is being developed to fill that gap.

“We are pleased to have received Orphan Drug Designation from the FDA for pamrevlumab in the treatment of DMD. There is high unmet medical need for patients suffering from this debilitating disease needing a new treatment option,” Elias Kouchakji, MD, senior vice president, Clinical Development and Drug Safety, for FibroGen, said in a press release.

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Pamrevlumab is currently being tested in an open-label Phase 2 clinical trial (NCT02606136) assessing its effectiveness and safety in non-ambulatory DMD patients at sites across the U.S. Twenty-one boys, age 12 or older, are enrolled and being treated with an intravenous (IV) infusion of pamrevlumab (35 mg/kg) every two weeks for a total of 156 weeks (three years). An interim analysis is expected after one year of treatment; depending on findings, enrollment may open to more patients.

The trial’s primary outcome is the annual change of forced vital capacity (FVC, the amount of air a patient is able to exhale after taking a deep breath) from baseline to week 104 (year two).

Secondary outcomes will include changes from study start (baseline) to year two of treatment in other measures of respiratory strength (e.g., forced expiratory volume, maximum inspiratory pressure), as well as muscle strength (e.g., grip strength, pinch strength) and tissue scarring (cardiac fibrosis score).

“All 21 non-ambulatory DMD patients in our ongoing phase 2 study with pamrevlumab have completed the first 52 weeks of treatment. We are evaluating a number of clinical parameters in this study, including lung function, cardiac function, and upper extremity muscle function, and tissue fibrosis. We look forward to the continued development of this investigational therapeutic,” Kouchakji said.

Orphan drug status to given to investigative treatments that aim to help people with a rare disease, which means disorders that affect fewer than 200,000 people in the U.S. The designation comes with benefits, including financial incentives for drug development and commercialization, U.S. market exclusivity for a period of seven years, FDA support for clinical studies, and special fee exemptions and reductions.

Pamrevlumab is also in clinical trials, and has been named an orphan drug and placed on fast track development by the FDA, as a potential treatment for IPF and pancreatic cancer.

Joana is currently completing her PhD in Biomedicine and Clinical Research at Universidade de Lisboa. She also holds a BSc in Biology and an MSc in Evolutionary and Developmental Biology from Universidade de Lisboa. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells — cells that make up the lining of blood vessels — found in the umbilical cord of newborns.
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Joana is currently completing her PhD in Biomedicine and Clinical Research at Universidade de Lisboa. She also holds a BSc in Biology and an MSc in Evolutionary and Developmental Biology from Universidade de Lisboa. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells — cells that make up the lining of blood vessels — found in the umbilical cord of newborns.
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3 comments

  1. Tejvanti Vinod Thakur says:

    Is this drug available in India as my son is suffering from DMD . Pls let me know the details. Thank you.. .

    • Joana Carvalho says:

      Hi Tejvanti,
      As far as I know, pamrevlumab hasn’t been approved in any country yet, because it is still being developed and tested in clinical trials (investigational drug).

  2. Sir I am brijesh Kumar Verma from up Barabanki suffering from muscular dystrophy, I am 27 years old, I am unable to walk for 4 years, will I be able to walk once again in my life or I will die due to muscular dystrophy, sir I am working in defence ministry at the post of auditor in PCDA (P) Allahabad I joined this post on 16 May in 2018 so I have completed 1 year continuous service and I have been given CGHS facility with that I can get the most costly treatment. SIR ANYONE PLEASE TELL ME HOW LONG IT WILL TAKE TO COMPLETE CURE OF MUSCULAR DYSTROPHY.

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