Kesimpta, a medication for the treatment of multiple sclerosis, underwent a rigorous process to gain approval from the U.S. Food and Drug Administration (FDA). The journey from the initial submission to the final approval involved several stages and took a significant amount of time. Below, we delve into the various aspects of Kesimpta's approval process, shedding light on the timeline and key milestones.
Development and Preclinical Testing
Before a drug can even be considered for FDA approval, extensive research and development are necessary. Pharmaceutical companies invest substantial resources and time in conducting preclinical studies on animals and in vitro to evaluate the drug's safety, efficacy, and potential side effects. These investigations help identify promising candidates for further evaluation.
• Preclinical research for Kesimpta involved testing its mechanism of action, dosage requirements, and potential toxicities across various animal models.
• Promising results from preclinical studies led to the initiation of clinical trials.
Clinical Trials: Phase 1, Phase 2, and Phase 3
Following successful preclinical studies, Kesimpta progressed to clinical trials involving human participants. Clinical trials assess the drug's safety, dosage, effectiveness, and potential side effects in a controlled environment. These trials follow a three-phase process:
Phase 1:
• A small group of healthy volunteers, generally not exceeding 100 individuals, is involved.
• The primary objective is to determine the drug's safety, dosage, and formulation.
Phase 2:
• A larger group, typically around a few hundred participants, who have the target disease is enrolled.
• The primary objective is to evaluate the drug's efficacy and side effects in treating the disease.
Phase 3:
• Involves a larger population, ranging from hundreds to thousands of participants.
• The primary objective is to confirm the drug's efficacy, monitor side effects, and compare it to existing treatments or placebos.
Kesimpta's Clinical Trials
As the clinical trials progress, Kesimpta's safety and efficacy were evaluated over a span of several years:
Phase 1:
• Kesimpta's Phase 1 clinical trial began in 2014 and involved over 100 healthy volunteers.
• The primary findings indicated the drug was safe and well-tolerated, leading to the initiation of Phase 2 trials.
Phase 2:
• Kesimpta's Phase 2 trials began in 2015 and included approximately 300 participants with multiple sclerosis.
• The results demonstrated the drug's efficacy in reducing relapse rates and improved overall patient outcomes.
Phase 3:
• Kesimpta's Phase 3 trial, known as ASCLEPIOS I and II, commenced in 2016 and involved over 1,800 participants.
• The results showed a significant reduction in annual relapse rates and disability progression compared to the standard treatment.
New Drug Application (NDA) Submission
Upon completion of the clinical trials, the pharmaceutical company collects and analyzes the data obtained during the studies. If the results demonstrate the drug's safety and efficacy, the company can submit a New Drug Application (NDA) to the FDA. The NDA comprises comprehensive data and information on the drug, including its manufacturing, labeling, and proposed indications.
FDA Review Process
Once the NDA is submitted to the FDA, it undergoes a detailed review process:
1. FDA Filings and Review Assignment:
• The FDA reviews the NDA submission for completeness.
• Upon acceptance, the application is assigned to a specific review division and team.
2. FDA Review by Division and Advisory Committee:
• An assigned review team examines the NDA, scrutinizing the data and supporting evidence.
• If necessary, an independent Advisory Committee evaluates the drug's safety and efficacy, providing recommendations.
3. Labeling and Post-Marketing Requirements:
• The FDA works with the pharmaceutical company to finalize the drug's labeling information.
• Post-marketing requirements, such as additional studies or safety monitoring, may be imposed.
Approval and Post-Approval Monitoring
After the FDA completes its review, it makes a decision regarding the drug's approval. If approved, the medication can be marketed, and the company must adhere to post-approval requirements:
Pricing:
• The pricing of Kesimpta can vary across different countries. As of June 2021, the approximate average annual list prices of Kesimpta in the United States, the United Kingdom, South Korea, Japan, and China are $105,000, £86,650, ₩190,000,000, ¥2,000,000, and ¥114,600, respectively.
Availability:
• Kesimpta became commercially available in the United States in August 2020, following its FDA approval.
• Availability in other countries may vary based on individual regulatory approval processes.
Post-Approval Monitoring:
• The FDA continues to monitor the medication's safety and effectiveness post-approval.
• Adverse events and any emerging safety concerns are closely monitored, and appropriate actions are taken if required.
Frequently Asked Questions (FAQs)
1. How long did it take for Kesimpta to receive FDA approval?
• The journey from the initiation of clinical trials to FDA approval took approximately 6 years.
2. What is the cost of Kesimpta in different countries?
• As of June 2021, the approximate average annual list prices of Kesimpta were $105,000 in the United States, £86,650 in the United Kingdom, ₩190,000,000 in South Korea, ¥2,000,000 in Japan, and ¥114,600 in China.
3. When did Kesimpta become available in the United States?
• Kesimpta became commercially available in the United States in August 2020, following FDA approval.
References:
1. FDA Approves Kesimpta® (ofatumumab), the First and Only Self-Administered, Targeted B-cell Therapy for Patients with Relapsing Multiple Sclerosis. (2020). Retrieved from [https://www.novartis.com/news/media-releases/fda-approves-kesimpta-ofatumumab-first-and-only-self-administered-targeted-b-cell-therapy-patients-relapsing-multiple-sclerosis](https://www.novartis.com/news/media-releases/fda-approves-kesimpta-ofatumumab-first-and-only-self-administered-targeted-b-cell-therapy-patients-relapsing-multiple-sclerosis)
2. ASCLEPIOS I and II Clinical Trials. (2019). Retrieved from [https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6783935/](https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6783935/)
