Description
The purpose of this guidance is to assist sponsors in the clinical development of drugs and biological products for the treatment of amyotrophic lateral sclerosis (ALS). Specifically, this guidance addresses the Food and Drug Administration’s (FDA’s) current thinking regarding the clinical development program and clinical trial designs for drugs to support an indication for the treatment of ALS.
Scope & Applicability
Product Classes
2Products requiring specific discussion with CBER; pharmacokinetic/pharmacodynamic considerations are different for these products
Requires analytical comparability per ICH Q5E
Stakeholders
1Entity responsible for submitting applications under section 524B
Regulatory Context
Attributes
3using biomarkers from early dose-finding studies to help identify dose
Criteria for responder analysis
Classification of diseases like ALS requiring specific effectiveness evidence.
Identified Hazards
Hazards
2intrinsic factor requiring potential dose adjustment
extrinsic factor requiring potential dose adjustment
Related CFR Sections (3)
- 21CFR314.105§ 314.105 Approval of an NDA and an ANDA.
(a) FDA will approve an NDA and send the applicant an approval letter if none of the reasons in § 314.125 for refusing to approve the NDA applies. FDA will issue a tentative approval letter if an NDA otherwise meets the requirements for approval under the Federal Food, Drug, and Cosmetic Act, but caRead full regulation →
- 21CFR314.125§ 314.125 Refusal to approve an NDA.
(a) The Food and Drug Administration will refuse to approve the NDA and for a new drug give the applicant written notice of an opportunity for a hearing under § 314.200 on the question of whether there are grounds for denying approval of the NDA under section 505(d) of the Federal Food, Drug, and CoRead full regulation →
- 21CFR312.80§ 312.80 Purpose.
The purpose of this section is to establish procedures designed to expedite the development, evaluation, and marketing of new therapies intended to treat persons with life-threatening and severely-debilitating illnesses, especially where no satisfactory alternative therapy exists. As stated § 314.10Read full regulation →
See Also (8)
- Duchenne Muscular Dystrophy and Related Dystrophinopathies: Developing Drugs for Treatment Guidance for Industry (Status: Final)
- Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products: Draft Guidance for Industry (Status: Draft)
- Nonclinical Testing of Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating or Life-Threatening Diseases Guidance for Sponsor-Investigators: Draft Guidance for Sponsor-Investigators (Status: Draft)
- Rare Diseases: Considerations for the Development of Drugs and Biological Products (Status: Final)
- Antibacterial Therapies for Patients With an Unmet Medical Need for the Treatment of Serious Bacterial Diseases (Status: Final)
- Rare Diseases: Early Drug Development and the Role of Pre-IND Meetings : Draft Guidance for Industry (Status: Draft)
- Limited Population Pathway for Antibacterial and Antifungal Drugs Guidance for Industry (Status: Final)
- Investigational New Drug Application Submissions for Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating or Life-Threatening Diseases: Chemistry, Manufacturing, and Controls Recommendations, Guidance for Sponsor-Investigators : Draft Guidance for Industry (Status: Draft)