“The U.S. Food and Drug Administration has awarded orphan drug status to a promising immunotherapy vaccine developed at Roswell Park Cancer Institute. The FDA notified MimiVax LLC, a Roswell Park spinoff company, on Aug. 3 that its application for orphan status for SurVaxM as treatment for glioblastoma, a type of brain cancer, had been approved.
“Orphan status is a special designation awarded to encourage innovation and exploration of approaches to treat rare diseases that affect relatively few people. SurVaxM, also known as DRU-2017-5947, is an immunotherapy drug that targets survivin, a cell-survival protein present in most cancers.”
“The FDA granted orphan drug designation to tucatinib for the treatment of patients with breast cancer whose disease metastasized to the brain, according to the drug’s manufacturer.
“Tucatinib (ONT-380, Cascadian Therapeutics) is an investigational, orally bioavailable, potent tyrosine kinase inhibitor that is highly selective for HER-2 without significant inhibition of EGFR, which has been associated with significant toxicities.”
“The FDA granted orphan drug designation to the WT1 cancer vaccine for the treatment of malignant pleural mesothelioma.
“The FDA based the designation on results from a randomized, double blind, placebo-controlled phase 2 trial of the WT1 vaccine (SLS-001, SELLAS Life Sciences) in 40 patients with malignant pleural mesothelioma treated at Memorial Sloan Kettering Cancer Center and The University of Texas MD Anderson Cancer Center.
“Results showed patients assigned the vaccine experienced longer median OS (21.4 months vs. 16.6 months) and median PFS (11.4 months vs. 5.7 months) than those assigned placebo. The vaccine also demonstrated a favorable safety profile, according to researchers.
“A phase 2b/3 trial in malignant pleural mesothelioma is expected to begin by the third quarter of this year.”
“The FDA granted orphan drug designation to IMCgp100 for the treatment of uveal melanoma.
“IMCgp100 (Immunocore) is part of a class of bispecific biologic reagents known as ImmTACs, or immune-mobilizing monoclonal T-cell receptors against cancer.
“The agents combine a T-cell receptor-based targeting system with an anti-CD3 effector function designed to activate a specific and potent T-cell response to recognize and destroy cancer cells, according to Immunocore.”
“The FDA granted orphan drug designation to MTG-201 for the treatment of patients with malignant mesothelioma, according to a press release.
“MTG-201 (MTG Biotherapeutics) is an advanced biologic therapy that targets Dickkopf-3 gene defects in various cancer types. The Dickkopf-3 gene produces the REIC protein, without which cancer cells can not die.
“MTG-201 directly attacks mesothelioma cancer cells and induces immune system response, and may be used in combination with anti–CTLA-4 therapies in development for the disease.
“In addition, MTG-201 is under development for the treatment of prostate cancer, and ongoing preclinical programs are evaluating MTG-201 as a potential treatment for liver and bladder cancers.”
“Aura Biosciences, a biotech company developing highly tumor-targeted breakthrough therapies for rare cancers, has been granted Orphan Disease Designation by the FDA for its drug AU-011 for the treatment of Uveal Melanoma. The FDA’s Orphan Drug Designation program provides orphan status to drugs and biologics, which demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions in the US. In addition, the first oral presentation of Aura Biosciences’ pre-clinical research, ‘Evaluating the in vivo efficacy of a first-in-class drug for the treatment of primary uveal melanoma’, was recently delivered by McGill University Health Centre researchers at the ARVO (Association for Research in Vision and Ophthalmology) Annual Meeting.
“ ‘There are currently no approved drug therapies for the treatment of uveal melanoma which is a rare but life threatening disease. We are thrilled to receive this Orphan Drug Designation that, together with the positive preclinical data, is enabling us to move this drug one step closer to the clinic,’ said Elisabet de los Pinos, founder and CEO of Aura Biosciences.”
“AstraZeneca today announced that the US Food and Drug Administration has granted Orphan Drug Designation for the anti-CTLA-4 monoclonal antibody, tremelimumab, for the treatment of malignant mesothelioma.
“Mesothelioma is a rare, aggressive cancer that most often affects the lining of the lungs and abdomen. Available treatments for mesothelioma are very limited, particularly for patients with advanced disease.
“ ‘There is a significant need for new treatment options for patients with mesothelioma because fewer than five percent of patients currently survive beyond five years, even when they receive timely diagnosis and care. Our aim is to rapidly advance the development of tremelimumab as a potential new treatment option for these patients,’ said Robert Iannone, Senior Vice President, Head of Immuno-oncology, Global Medicines Development at AstraZeneca.
“The Orphan Drug Designation programme provides orphan status to drugs and biologics, which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the US1.”
The gist: The U.S. Food and Drug Administration (FDA) has granted “orphan drug” designation to a new drug called vs-5584 (Verastem) for mesothelioma. The designation will make it easier for the drug maker to successfully develop Verastem and get it to patients in the U.S. Verastem has shown promise in patients in a clinical trial.
“The FDA granted orphan drug designation to VS-5584 for the treatment of mesothelioma, according to a press release from the drug’s manufacturer.
“VS-5584 (Verastem) has demonstrated potent and highly selective activity against class 1 PI3K enzymes and dual inhibitory actions against mTORC1 and mTORC2, according to a press release issued by Verastem.
“ ‘This is an important regulatory milestone for Verastem and, together with our European orphan medicinal product designation, will facilitate our global development of VS-5584 to help improve the available treatment options for patients suffering from this highly aggressive cancer,’ Robert Forrester, president and CEO of Verastem, said in a press release. ‘We look forward to taking full advantage of the opportunities that orphan designation allows in order to bring this potential new treatment option to patients as rapidly as possible.’ ”