Partner Therapeutics, Inc. (PTx), a commercial biotechnology company, announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Leukine® (sargramostim), a yeast-derived recombinant human granulocyte-macrophage colony stimulating factor (rhuGM-CSF), for the potential treatment of Stage IIb-IV melanoma.
LEXINGTON, Mass., Sept. 19, 2019 /PRNewswire/ -- Partner Therapeutics, Inc. (PTx), a commercial biotechnology company, announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Leukine® (sargramostim), a yeast-derived recombinant human granulocyte-macrophage colony stimulating factor (rhuGM-CSF), for the potential treatment of Stage IIb-IV melanoma. Melanoma is the most aggressive form of skin cancer and rates of melanoma have been rising for the past 30 years. The American Cancer Society estimates 96,480 new melanoma cases will be diagnosed in the US and 7,230 people will die from the disease in 2019. The FDA grants orphan drug designation to promote the development of promising treatments for conditions that affect 200,000 or fewer U.S. patients annually. If a product holding Orphan Drug Designation receives the first FDA approval for the disease in which it has such designation the company qualifies for, among other things, seven years of market exclusivity following marketing approval. The Eastern Cooperative Oncology Group (ECOG) previously reported results of Study 1608, a Phase II study in which patients with advanced stage melanoma received a combination of sargramostim and ipilimumab or ipilimumab alone1. Among 245 patients, the addition of sargramostim led to longer survival (median 17.5 vs 12.7 months). The most common Grade 3-5 toxicities in patients treated with sargramostim and ipilimumab were diarrhea (12.7%) and rash (9.3%) and occurred at similar rate in patients receiving ipilimumab alone. Overall, severe toxicities occurred less frequently in patients treated with sargramostim plus ipilimumab vs ipilimumab alone (44.9% vs 58.3%, Grade 3-5); the most notable reductions were in gastrointestinal and pulmonary toxicities. The results of this study led to initiation of a larger Phase 2/3 study (ECOG 6141) evaluating sargramostim in combination with ipilimumab and nivolumab as initial treatment of advanced or metastatic melanoma. This ongoing study is being conducted by ECOG with support from the National Cancer Institute.2 “Leukine’s role as an immunomodulator was not the initial focus when it was first discovered decades ago. As we learn more about the immunologic effects of GM-CSF on antitumor immunity, we believe there is potential to develop Leukine to help more patients benefit from treatment with checkpoint inhibitors in melanoma and other difficult to treat cancers,” said Bob Mulroy, PTx’s CEO. “This orphan designation is an important step in the development of Leukine. We are pleased FDA has programs such as Orphan Drug Designation to support research in rare diseases.” Leukine was initially approved in the United States in 1991 and has five hematologic oncologic indications. Leukine is currently not approved for the treatment of melanoma. The approval of an orphan drug designation request does not alter the standard regulatory requirements and processes for obtaining marketing approval of an investigational drug. Sponsors must establish safety and efficacy of a compound in the treatment of a disease through adequate and well-controlled studies. Please see full Prescribing Information for LEUKINE® at www.leukine.com About Leukine® (sargramostim) Leukine® is a yeast-derived recombinant humanized granulocyte-macrophage colony stimulating factor (rhuGM-CSF) and the only FDA approved GM-CSF. GM-CSF is an important leukocyte growth factor known to play a key role in hematopoiesis, effecting the growth and maturation of multiple cell lineages as well as the functional activities of these cells in antigen presentation and cell mediated immunity3. Important Safety Information for LEUKINE® (sargramostim) Contraindications
Warnings and Precautions
Adverse Reactions Adverse events occurring in >10% of patients receiving LEUKINE® in controlled clinical trials and reported in a higher frequency than placebo are:
Please see full Prescribing Information for LEUKINE® at www.leukine.com Indications and Usage LEUKINE® (sargramostim) is a leukocyte growth factor indicated for the following uses:
About Partner Therapeutics, Inc.: PTx is an integrated commercial-stage biotech company focused on the development and commercialization of therapeutics that improve health outcomes in the treatment of cancer. PTx’s development focus spans the entire range of cancer therapy from primary treatments to supportive care. The company believes in delivering great products with the purpose of creating the best possible outcomes for patients and their families. References: 2. National Cancer Institute. Nivolumab and ipilimumab with or without sargramostim in treating patients with stage III-IV melanoma that cannot be removed by surgery. Available at: https://clinicaltrials.gov/ct2/show/NCT02339571. NLM identifier: NCT02339571. Accessed August 26, 2019 3. Armitage JO. Blood. 1998;92(12):4491-4508
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