In the world of rare disease research, the path to developing new treatments is often fraught with challenges. The small patient populations, limited commercial incentives, and high costs of traditional drug development can result in a scarcity of therapeutic options. However, drug repurposing is changing the game for rare disease patients. By finding new uses for existing drugs, researchers are uncovering life-changing therapies faster and more cost-effectively than ever before. Fortuity Pharma’s work to repurpose amlexanox is a prime example of this transformative strategy.
What is Drug Repurposing?
Drug repurposing involves taking an existing drug—approved for at least one condition—and identifying its potential to treat a different condition. This approach capitalizes on the extensive safety, mechanism of action and effectiveness data already available for the drug, significantly accelerating the path to clinical trials and regulatory approval.
For rare diseases, where the unmet medical need is substantial, drug repurposing offers an opportunity to address therapeutic gaps without starting from scratch. This strategy is particularly appealing because it:
- Reduces Development Costs: Developing a new drug can cost over $1 billion and take 10-15 years. Repurposing bypasses the early discovery and safety phases, cutting both time and costs.
- Speeds Up Timelines: Drugs with known safety profiles can often enter clinical trials directly in the target population, shaving years off the traditional development timeline.
- Decreases Risk: The known safety profiles of repurposed drugs reduce the likelihood of unexpected adverse effects, increasing the probability of success in trials.
- Broadens Access: By focusing on off-patent drugs, repurposing efforts can create affordable treatment options, ensuring accessibility for underserved rare disease communities.
The Potential of Amlexanox
Amlexanox’s journey exemplifies the promise of drug repurposing. Originally developed in Japan as a treatment for asthma and later approved for use in the U.S. to treat aphthous ulcers (canker sores), amlexanox fell out of use when newer therapies emerged. However, its mechanism of action—specifically, its ability to suppress nonsense-mediated decay (NMD) and promote readthrough of nonsense mutations—has opened new possibilities for rare diseases caused by genetic mutations.
Rare conditions, such as WOREE Syndrome and other disorders have some individuals where their disease is driven by nonsense mutations, result in premature stop codons that truncate essential proteins. Amlexanox has shown potential in preclinical studies to bypass these faulty genetic instructions, restoring protein function. Furthermore, its ability to cross the blood-brain barrier makes it particularly promising for addressing central nervous system (CNS) symptoms in rare diseases.
Amlexanox and Fortuity Pharma’s Mission
At Fortuity Pharma, we are committed to bringing amlexanox back to the market to address the unmet needs of rare disease patients. Our focus is on leveraging its established safety profile and unique mechanisms to develop treatments for conditions with no current options. This includes collaborating with patient groups, researchers, and clinicians to identify diseases where amlexanox’s therapeutic potential is greatest.
The story of Lucia, a child with WOREE Syndrome whose remarkable progress on amlexanox provided her family with unprecedented hope, underscores the urgency of this work. Though amlexanox was discontinued by its original manufacturer due to commercial reasons, our efforts to revive it are a testament to the power of drug repurposing in creating new opportunities from existing science.
The Broader Impact of Drug Repurposing
Drug repurposing isn’t just a strategy for rare diseases; it’s a paradigm shift for the entire pharmaceutical industry. By thinking creatively about existing drugs, researchers can:
- Address Neglected Diseases: Repurposed drugs are increasingly used for conditions like tuberculosis, malaria, and other diseases with limited commercial appeal.
- Enhance Personalized Medicine: By understanding how drugs work in diverse genetic contexts, repurposing can lead to more targeted, effective treatments.
- Expand Research Opportunities: Every repurposed drug sheds light on new pathways and mechanisms, fueling further innovation.
A Call to Action
Drug repurposing is a powerful tool, but its success relies on collaboration. We need more partnerships between academia, industry, patient advocacy groups, and regulatory agencies to identify and advance repurposing opportunities. At Fortuity Pharma, we are proud to be part of this growing movement, demonstrating how innovative thinking can translate into real-world impact.
By focusing on repurposing amlexanox for rare diseases, we aim to bring hope and healing to patients who have waited too long for effective treatments. Together, we can turn yesterday’s drugs into tomorrow’s cures.
