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The FDA Has Granted Fast Track Designation to ANPD001, an Investigational Autologous Cell Therapy for the Treatment of Parkinson's Disease

Friday, October 20, 2023

Aspen Neuroscience recently announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for their ANPD001 treatment for Parkinson's disease (PD), aimed at improving motor function. ANPD001 is a personalized (autologous) cell therapy designed to treat PD by replacing lost dopamine neurons, and it is developed from the patient's own cells.

The Fast Track Designation is a special status given by the FDA to expedite the development and review of new drugs addressing serious medical conditions with unmet needs. It offers several advantages, such as early and frequent interactions with the FDA during clinical development, potential for accelerated approval, and priority review.

Aspen, expressed their satisfaction with the FDA's Fast Track Designation, emphasizing the potential of ANPD001 as a valuable treatment option for individuals with Parkinson's disease. This designation represents a significant step towards collaborating with the FDA and advancing the development of this therapy.

Aspen, further emphasized their commitment to bringing ANPD001 to patients safely and expeditiously.

Aspen plans to initiate a Phase 1/2a clinical trial of ANPD001 for patients with moderate to severe PD, following their Trial-Ready Screening Cohort Study in 2022, which screened, enrolled, and began manufacturing cells for potential participants. This trial will be the first multicenter Phase 1/2a trial of an autologous iPSC-derived therapy in the United States.

Aspen's approach involves using induced pluripotent stem cells (iPSCs), generated from the patient's own skin cells, to create dopamine neuronal precursor cells (DANPCs). These cells undergo rigorous quality control assessments, including Aspen's proprietary artificial intelligence-based genomics tests, before being implanted in the patient.

ANPD001 is an experimental cell therapy product being investigated as an autologous neuron replacement for Parkinson's disease. Aspen's process begins with a small sample of the patient's skin cells, which are reprogrammed into iPSCs and then differentiated into DANPCs. These cells are surgically provided to the patient to replace those lost or damaged due to the disease, with their quality assessed at every manufacturing stage using Aspen's proprietary artificial intelligence-based genomics tests.

The Phase 1/2a clinical trial for ANPD001 is a single-arm, open-label study that will evaluate the safety, tolerability, and preliminary efficacy of ANPD001 in patients with moderate to severe PD through planned dose-escalation.

Parkinson's disease is the second most common neurodegenerative disease worldwide. It can manifest as non-motor symptoms, including mental and behavioral changes, sleep disturbances, depression, memory issues, and fatigue, as well as motor symptoms caused by the loss of dopamine neurons in the brain. These motor symptoms include unintended movements, shaking, stiffness, and difficulties with balance and coordination, which worsen over time. At the time of diagnosis, many individuals have already lost a significant number of dopamine-producing cells in the brain. Currently, there is no disease-modifying therapy available that can halt, replace, or slow down the progression of Parkinson's disease.



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