For Patients

Empowering Those Affected with Affordable Therapies

Understanding Potential Treatments from Fortuity Pharma: Our Commitment to Those Affected

The team at Fortuity Pharma brings life-changing therapies to those affected by rare disease by finding new rare disease uses for existing drugs. We understand the unique challenges of rare diseases, and our mission is to prove that existing drug treatments like amlexanox can be safely and affordably repurposed and made available to those who need them.

How Amlexanox Might Help

Amlexanox was originally approved as a chronic treatment for adults and children asthma in 1987. It worked because it reduced inflammation, and those taking it had good results and few side effects.

Amlexanox has shown promise in research studies as a potential therapy for a specific sub-population of those affected by rare gene disorders such as WOREE syndrome, cystic fibrosis, Duchenne muscular dystrophy, Charcot-Marie-Tooth disease, aspartylglucosaminuria, and epidermolysis bullosa. Genes are like messages that are read, and the reading mechanism normally uses the message to build a functioning protein.

Some people have a gene that doesn’t work properly because one “letter” in the gene message is the wrong letter. Sometimes, this wrong letter, called a nonsense mutation, functions like a period, and prematurely stops the mechanism that “reads” the gene. Because the reading stops, the message is too short, and the protein either never gets made, or is too short to do its job. So those with a nonsense mutation don’t have enough protein, which leads to symptoms.

Restoring the Full Protein

Amlexanox just happens to have the right size and shape to fit into the gene “reading mechanism” and tell it to ignore the false period. So the whole gene message gets read, and because of this, a full length protein gets made, and can partially or fully restore the function caused by the gene defect.

Through ongoing lab testing and clinical trials, we are working to prove it will help those with rare diseases who have this nonsense mutation genotype and then ensure it’s available as soon as possible.

How to Know if You Have a Nonsense Mutation

Most genetic rare diseases can have multiple gene mutations. For most diseases, about of the gene mutations are nonsense mutations. Amelxanox can potentially help with those. It is unlikely that amlexanox will help with missense mutations, insertions, deletions and other gene mutations. The best way to know what type of mutation you have is to have genetic testing completed and the results explained by a genetic counselor.

Unfortunately, at this time Fortuity Pharma does not have the capability to help with this genetic testing or counselling. Your clinician or patient advocacy group should be able to advise you.

How to Get Involved

Are you or your loved one affected by a rare genetic condition that could be caused by a nonsense mutation? Are you involved with a patient advocacy group? We would be eager to hear from you or your advocacy group so we can determine if our work on amlexanox might be of value. Please contact us directly for more information.

For Clinicians