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Summary:
This press release announces that Veloxis pharmaceuticals has received Orphan Drug Designation from the FDA for its drug candidate, pegrizeprument (also known as VEL-101 and FR104). This designation is for the prevention of acute rejection in solid organ transplant recipients, specifically liver transplant (OLT) recipients. the company believes this drug has the potential to address a significant unmet medical need in transplant patients, as current immunosuppressant options have limitations. Veloxis licensed the drug from OSE Immunotherapeutics in 2021 and is actively researching its potential. Veloxis is a subsidiary of Asahi Kasei.
Key takeaways:
* Drug: Pegrizeprument (VEL-101, FR104)
* Company: Veloxis Pharmaceuticals, Inc. (an Asahi Kasei company)
* Designation: FDA Orphan Drug Designation
* Indication: Prevention of acute rejection in solid organ transplant recipients (focus on liver transplants)
* Mechanism of Action: Blocks CD28-mediated T-cell activation while allowing CTLA-4 mediated immunosuppression. This is a dual-action approach.
* Unmet Need: Current immunosuppressants have drawbacks, and there’s a need for better therapies to prevent rejection without compromising patient health.
* Licensing: Veloxis acquired worldwide rights to develop and commercialize pegrizeprument from OSE Immunotherapeutics in April 2021.
Detailed Information (organized by section):
* Problem: Graft rejection is a serious complication of organ transplantation, impacting both the graft and the patient’s survival.
* Orphan drug Designation: Granted for drugs targeting rare diseases (affecting fewer than 200,000 people in the US). It incentivizes the growth of treatments for these conditions.
* Pegrizeprument Details:
* It’s a pegylated monoclonal antibody fragment.
* It blocks CD28, a costimulatory molecule involved in T-cell activation.
* Crucially, it doesn’t block CTLA-4, which is a natural immune regulator. This is intended to provide a more balanced immunosuppressive effect.
* Company Information:
* Veloxis: Focused on improving the lives of transplant patients through the development and commercialization of medications.Based in Cary, North Carolina.
* Asahi Kasei: A larger Japanese company that owns Veloxis. It’s a diversified company with healthcare, home, and materials businesses.
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