Stealth BioTherapeutics is looking to put its tumultuous path behind it with a new name.
That new name is mighty—literally, as the biopharma is now Mighty Therapeutics, reflecting its recent change from being an R&D-focused company to now a commercial biopharma as well, the company said in a June 16 release.
Last September, what was then Stealth nabbed an FDA treatment nod for the ultrarare disease Barth syndrome, marking the peptide as the first therapy for the condition, and Stealth’s first marketed med.
Stealth’s injection, known as elamipretide, is now sold as Forzinity for the treatment of Barth syndrome, an X-linked genetic disorder that weakens the heart and other muscles. Death rates are highest in the first four years of life, and patients don’t typically live past their 40s.
At the end of April, the company said in a corporate update that 33 patients have started Forzinity therapy with 85% of patients fully covered, “reflecting meaningful early adoption across treatment centers.” It did not give any financial details of the launch.
The path to approval was anything but smooth, however. The FDA rejected the drug twice over several years, which eventually led to a public outcry and the intervention of 14 members of Congress.
They urged then-FDA Commissioner Marty Makary, M.D., to provide clarity about the approval process for Forzinity before the medicine eventually got the green light.
Stealth also questioned its own viability after one of the rejections, laying off 30% of staffers in an effort to save cash right after receiving a complete response letter.
The reprieve of last year’s approval now leads to a new chapter that clearly the company expects to be big.
On top of its approval, the company also has a pipeline of experimental drugs seeking to help correct mitochondrial dysfunction underlying a range of rare and age-related diseases, including dry AMD and Leigh syndrome.
“When I hear the word ‘Mighty,’ I think about continuing to push forward even when things are hard,” said Walker Burger, an individual living with Barth syndrome, quoted in Mighty’s release.
“Living with Barth syndrome takes strength every day,” Burger added. “To see a company choose a name that reflects how strong this community is makes me feel seen and hopeful for what comes next.”