Expanded Access Policy
CAELUM BIOSCIENCES EXPANDED ACCESS POLICY FOR INVESTIGATIONAL DRUG CAEL-101 (Chimeric fibril-reactive monoclonal antibody 11-1F4 (Ch11-1F4)) FOR TREATMENT OF LIGHT CHAIN AMYLOIDOSIS
Caelum Biosciences, Inc. (“Caelum”) is a clinical-stage biotechnology company developing CAEL-101, a novel antibody, in collaboration with Alexion Pharmaceuticals for the treatment of adult patients with amyloid light chain amyloidosis. Under the 21st Century Cures Act, the manufacturer or distributor of one or more investigational drugs for the treatment of one or more serious diseases or conditions is required to make available its policy on how it evaluates and responds to requests submitted under section 561(b) of the Federal Food, Drug, and Cosmetic Act for provision of such a drug. Described below is Caelum’s policy for reviewing and responding to requests for individual patient access to our investigational drug CAEL-101 antibody for the treatment of amyloid light chain amyloidosis.
CAEL-101 is under investigation for the treatment of amyloid light chain amyloidosis. The benefits and risks of CAEL-101 have not yet been established. Caelum believes that CAEL-101 should be available and studied in patients as part of a randomized-controlled clinical trial program to obtain sufficient data on the safety and efficacy of CAEL-101 that may be used to support approval of the product and subsequent wider accessibility to patients with light chain amyloidosis. Therefore, Caelum does not offer an Expanded Access Program at this time. We encourage patients with light chain amyloidosis to speak with their physicians and consider participating in clinical trials. Caelum will review this policy from time-to-time to determine if wider access to CAEL-101 is appropriate through an Expanded Access Program, based on available safety and efficacy data.
A treating physician may submit questions or requests regarding expanded access for CAEL-101 to the following: firstname.lastname@example.org
Additional information on Expanded Access may be obtained from the U.S. Food and Drug Administration at: https://www.fda.gov/news-events/expanded-access/expanded-access-information-physicians
The clinical trial record containing information on the current protocols is available on clinicaltrials.gov:
As authorized by the 21st Century Cures Act, Caelum may revise this expanded access policy at any time. Additionally, the posting of the policy or an expanded access protocol by Caelum shall not serve as a guarantee of access to CAEL-101 and/or enrollment into the protocol for any patient.