Participants for START Pilot Program Selected by FDA

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The seven chosen sponsors will help accelerate development of novel drugs and biologics for rare diseases.

FDA has chosen seven companies to participate in its Support for clinical Trials Advancing Rare disease Therapeutics (START) Pilot Program. START was created to advance development of novel drugs and biologics for rare diseases. The pilot will gather information on how to best facilitate efficient development of therapies for rare diseases and assist sponsors to generate high-quality data to support new drug or biologics license applications.

The Center for Drug Evaluation and Research (CDER) chose the following companies to participate in START for the development of rare neurodegenerative conditions:

  • Larimar Therapeutics to treat Friedreich’s ataxia through its Nomlabofusp product
  • Calico Life Sciences to treat vanishing white matter disease with its ABBV-CLS-7262 product
  • Denali Therapeutics for its DNL 126 product to treat Mucopolysaccharidosis Type IIIA (Sanfilippo syndrome).

The Center for Biologics Evaluation and Research (CBER) chose the following companies for their gene or cellular therapies to treat rare diseases:

  • Grace Science for their GS-100 (AAV0-rhNGLY1) product to treat NGLY1 deficiency
  • Moderna TX for their mRNA-3705 product to treat isolated methylmalonic acidemia due to complete or partial methylmalonyl-coenzyme A mutase deficiency
  • Myrtelle to treat Canavan disease with their product, rAAV-Olig001-ASPA
  • Neurogene for the treatment of Rett syndrome with NGN-401.

“Although there were a number of excellent and qualified applicants, there was only availability for a limited number of programs within the START Pilot Program. In making these selections, we considered the strength of the development programs, and the unmet medical need represented by the indicated conditions. To help aid our evaluation of the program, we also attempted to include a spectrum of entities in the pilot ranging from companies initially established to produce a single product to established biotechnology companies,” CDER Director, Patrizia Cavazzoni and CBER Director, Peter Marks stated (1).

Participants in START will receive advice from FDA regarding product-specific development issues such as clinical study design, control groups, and patient populations. Early development problems will be addressed to avoid potential delays or prevention of trial progression. Access to FDA programs, such as the Advancing Real-World Evidence Program, Complex Innovative Trial Design program, Translational Science Team, and Rare Disease Endpoint Advancement Pilot, will be given to those participating in START.

Eligibility requirements to participate in START per FDA include (2):

  • “For both CBER and CDER, the program is open to sponsors of products currently in clinical trials under an active investigational new drug application (IND) and IND has been submitted in or converted to Electronic Common Technical Document (eCTD) format, unless the IND is of a type granted a waiver from eCTD format.”
  • “For both CBER and CDER, the program is open to sponsors who have demonstrated substantial effort to ensure that Chemistry, Manufacturing, and Controls (CMC) development aligns with clinical development.”
  • “For CBER, eligible products must be a gene or cellular therapy intended to address an unmet medical need as a treatment for a serious rare disease or condition, which is likely to lead to significant disability or death within the first decade of life.”
  • “For CDER, products must be intended to treat rare neurodegenerative conditions, including those of rare genetic metabolic etiology.”

“CBER and CDER look forward to actively working together to fulfill our shared vision of bringing better treatment options to patients and their families. Through the START Pilot Program, the opportunity for increased engagement with the selected sponsors should facilitate more rapid achievement of regulatory milestones, if supported by the data and the science,” Cavazzoni and Marks stated (1).


  1. FDA. FDA Opens Doors for More Treatments for Rare Diseases through the New START Pilot Program. June 27, 2024.
  2. FDA. Support for clinical Trials Advancing Rare disease Therapeutics (START) Pilot Program. Updated June 27, 2024.