Health and Human Services Department, Food and Drug Administration
The Food and Drug Administration (FDA, Agency, or we) is announcing the availability of a draft guidance for industry entitled "Myelodysplastic Syndromes: Developing Drug and Biological Products for Treatment." The purpose of this guidance is to assist sponsors in the clinical development of drug and biological products for the treatment of the myelodysplastic syndromes (MDS). Specifically, this guidance addresses FDA's current thinking regarding the overall development program and clinical trial designs for the development of drug and biological products to support an indication of treatment of MDS. This guidance will focus specifically on development of drug and biological products that are considered disease-modifying and, therefore, will not cover products considered supportive only (e.g., erythropoiesis-stimulating agents). Furthermore, the guidance will not address drug development for MDS/myeloproliferative neoplasms, such as chronic myelomonocytic leukemia, which are considered a separate class of myeloid neoplasms.
Document Headings Document headings vary by document type but may contain the following: the agency or agencies that issued and signed a document the number of the CFR title and the number of each part the document amends, proposes to amend, or is directly related to the agency docket number / agency internal file number the RIN which identifies each regulatory action listed in the Unified Agenda of Federal Regulatory and Deregulatory Actions See the Document Drafting Handbook for more details. Department of Health and Human Services Food and Drug Administration [Docket No. FDA-2025-D-0649] AGENCY: Food and Drug Administration, HHS. ACTION: Notice of availability. SUMMARY: The Food and Drug Administration (FDA, Agency, or we) is announcing the availability of a draft guidance for industry entitled “Myelodysplastic Syndromes: Developing Drug and Biological Products for Treatment.” The purpose of this guidance is to assist sponsors in the clinical development of drug and biological products for the treatment of the myelodysplastic syndromes (MDS). Specifically, this guidance addresses FDA's current thinking regarding the overall development program and clinical trial designs for the development of drug and biological products to support an indication of treatment of MDS. This guidance will focus specifically on development of drug and biological products that are considered disease-modifying and, therefore, will not cover products considered supportive only ( e.g., erythropoiesis-stimulating agents). Furthermore, the guidance will not address drug development for MDS/myeloproliferative neoplasms, such as chronic myelomonocytic leukemia, which are considered a separate class of myeloid neoplasms. DATES: Submit either electronic or written comments on the draft guidance by September 2, 2025 to ensure that the Agency considers your comment on this draft guidance before it begins work on the final version of the guidance. ADDRESSES: You may submit comments on …
Citation: 90 FR 29014
