This week on The CheckRare Brief, we are joined by Eugene Lee, Head of CMI Media Group’s Rare Disease Division, for an industry perspective on some of the biggest developments in rare disease.
New Rare Disease Center of Excellence
CMI Media Group recently launched a Specialized Rare Disease Center of Excellence to scale precision marketing to smaller pharmaceutical companies. CMI is well known for its media and communications capabilities in the healthcare space for 37 years. CMI is now applying this longstanding commitment to the healthcare industry to rare conditions, which require a more targeted approach because patient populations and HCP audiences are smaller and highly specialized. CMI’s Centers of Excellence (COE) bring together CMI’s data, technology, expertise, and experience to help clients navigate that complexity.
The FDA approved Bravnesta (lutetium Lu 177 dotatate) to treat neuroendocrine disorders.
The approval is for a generic version of Novartis’ radioligand therapy, Lutathera, to treat somatostatin receptor-positive gastroenteropancreatic neuroendocrine tumors (GEP-NETs). The approval is largely based on showing the new generic drug, Bravnesta, has similar bioequivalence and therapeutic equivalence.
Novartis’ therapy has been available since 2018 and, in that time, has provided great benefit to these patients, and those patients now have a generic option to assist them.
The FDA also approved Lyrfigtu (lirafugratinib) as second-line therapy for patients with cholangiocarcinoma.
Cholangiocarcinoma is an aggressive cancer of the bile duct. Standard first-line therapy for the past 10 years has been gemcitabine and cisplatin, a chemotherapy combination used for many types of cancers. If that treatment is no longer effective, Lyrfigtu is an option.
Lyrfigtu is a FGFR2 inhibitor, and FGFR2 is a protein often altered in cancers. The drug is also being tested for other solid tumors with FGFR2 fusions. The data supporting approval came from a phase 1/2 study in which median progression-free survival was 11 months. Which, for this aggressive cancer, is very significant.
Second-line therapies, especially for rare cancers, are a common initial indication for new orphan drugs. First-line treatments are often well established, as is the case here, so replacing them requires a strong body of evidence.
Finally, the FDA issued new guidelines for clinical trials involving rare diseases, and other diseases with diverse patient populations.
The new initiative is a statistical model that accounts for different symptoms and presentations in patients with the same disease. For example, in Fabry disease, some have poor kidney function, others have cardiac problems, others have neuropathic pain. To have a clinical trial that only include one of those symptoms as the baseline outcome measure, limits the patients who can enrol.
The new FDA model allows patients to enroll with different baseline levels for different outcome measures and subgroup them accordingly, but the final analysis combines those subgroups into a total group to see whether the drug is effective, statistically speaking. It can also reduce the recruitment time for these small rare conditions from several years to several months.
The rare disease space is known for its innovative research and it is nice to see that innovation also occurring at the regulatory level.
Looking ahead
Several important FDA decisions are coming up, including:.
- Elevar Therapeutics’ therapy for cholangiocarcinoma and Praxis’ therapy for SCN2A and SCN8A deficiencies.
- Egetis Therapeutics’ orphan drug to treat patients with MCT8 deficiency.
References
CMI Media Group Launches Specialized Rare Disease Center of Excellence (COE) to Scale Precision Marketing for Niche Pharma
FDA approves Lu177 dotatate for NETs
FDA approves drug for 2nd line cholangiocarcinoma
FDA Provides novel ways to design clinical trials for rare diseases while still be statistically compliant