FDA grants orphan drug designation to several oncology therapies
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The FDA granted orphan drug designation to several therapies in development for oncology indications.
The proposed indications for these therapies include Waldenstrom macroglobulinemia, multiple myeloma, lung cancer and brain cancer.
Here is a summary of each agent:
MB-106 (Mustang Bio) is a CD20-targeted, autologous chimeric antigen receptor T-cell therapy. The orphan drug designation applies to use of the agent for treatment of Waldenstrom macroglobulinemia.
Paxalisib (Kazia Therapeutics Limited) is a phosphatidylinositol 3-kinase inhibitor. The designation applies to use of the agent for treatment of atypical rhabdoid/teratoid tumors, a rare and aggressive childhood brain cancer.
VBI-1901 (VBI Vaccines) is a bivalent gB/pp65 immunotherapeutic vaccine candidate. The designation applies to use of this agent for the treatment of glioblastoma.
Ilixadencel (Immunicum AB) is an off-the-shelf cell-based cancer immunotherapy developed for the treatment of solid tumors. The designation applies to use of the agent as treatment for gastrointestinal stromal tumors.
QXL138AM (Nammi Therapeutics) is a masked immunocytokine that targets a masked interferon alpha to the CD138 protein on the surface of the tumor cells. The designation applies to use of the agent for treatment of multiple myeloma.
PT217 (Phanes Therapeutics Inc.) is a bispecific antibody that targets delta-like ligand 3 and CD47. The designation applies to use of the agent for treatment of small cell lung cancer.
The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.
References:
- Mustang Bio announces orphan drug designation granted to MB-106, a CD20-targeted, autologous CAR T cell therapy for the treatment of Waldenstrom macroglobulinemia (press release). Available at: https://ir.mustangbio.com/news-events/press-releases/detail/145/mustang-bio-announces-orphan-drug-designation-granted-to. Published June 22, 2022. Accessed June 30, 2022.
- VBI Vaccines receives U.S. FDA orphan drug designation for VBI-1901 for the treatment of glioblastoma (press release). Available at: https://www.vbivaccines.com/press-releases/fda-orphan-drug-designation-vbi-1901-gbm/. Published June 22, 2022. Accessed June 30, 2022.
- U.S. FDA awards orphan drug designation (ODD) to paxalisib for AT/RT, a rare form of childhood brain cancer (press release). Available at:https://www.kaziatherapeutics.com/site/PDF/919bfdb2-7172-4d18-9474-6a52d67995e5/FDAAWARDSORPHANDESIGNATIONTOPAXALISIBFORATRT. Published June 17, 2022. Accessed June 30, 2022.
- Immunicum received FDA orphan drug designation for ilixadencel as treatment of gastrointestinal stromal tumors (GIST) (press release). Available at: https://mendus.com/news/immunicum-ab-immunicum-received-fda-orphan-drug-designation-for-ilixadencel-as-treatment-of-gastrointestinal-stromal-tumors-gist. Published June 9, 2022. Accessed June 30, 2022.
- Nammi Therapeutics granted FDA orphan drug designation for QXL138AM for the treatment of multiple myeloma (press release). Available at: https://www.prnewswire.com/news-releases/nammi-therapeutics-granted-fda-orphan-drug-designation-for-qxl138am-for-the-treatment-of-multiple-myeloma-301566764.html. Published June 13, 2022. Accessed June 30, 2022.
- Phanes Therapeutics’ PT217 receives orphan drug designation for small cell lung cancer from the FDA (press release). Available at: https://www.phanesthera.com/news/phanes-therapeutics-pt217-receives-orphan-drug-designation-for-small-cell-lung-cancer-from-the-fda/. Published June 21, 2022. Accessed June 30, 2022.
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