The FDA’s recent green light for Agamree (vamorolone) in the treatment of Duchenne muscular dystrophy (DMD) has ignited a wave of excitement at Santhera. This milestone is not just a standalone achievement but serves as a launching pad for future possibilities.
Santhera, a Swiss pharmaceutical company, is now eagerly anticipating the myriad of conditions that the novel corticosteroid vamorolone could potentially address. This unique dissociative steroid has been designed to retain the therapeutic efficacy of traditional steroids while evading the often debilitating side effects associated with them. According to Santhera, this groundbreaking mechanism of action positions vamorolone as a potential solution for various inflammatory disorders currently managed by steroids, but which can leave patients grappling with undesirable side effects.
Shabir Hasham, M.D., Santhera’s Chief Medical Officer, explained in an interview, “There’s a list of things that—as longer-term data comes out—we’re hoping to come to market with and say, ‘Here’s a molecule that gives you the anti-inflammatory benefits of steroids, which are very well documented in Duchenne, but because it’s better tolerated and because we avoid some of these life-changing side effects, you can actually stay on a higher dose for longer.'”
The spectrum of conditions vamorolone could potentially address encompasses asthma, inflammatory bowel disease, rheumatoid arthritis, multiple sclerosis, and various rare genetic diseases like DMD, prioritizing rare diseases as the initial focus of Santhera’s development efforts.
Dario Eklund, Santhera’s CEO, emphasized that one of the most significant advantages of this approval is the funding it provides for exploring Agamree’s potential. He noted, “By the time I’m retired and looking back at this venture, I don’t think that Duchenne will be the biggest indication generating revenue for the drug. I think there’s going to be a lot of other indications and bigger indications.”
Catalyst Pharmaceuticals holds the commercial rights for Agamree in North America, having paid $231 million, including an upfront payment of $90 million, in June. This deal enabled Santhera to settle its debts and extend its financial runway until the first quarter of 2025. Catalyst plans to launch Agamree in the first quarter of the following year.
Santhera’s focus has now shifted to Europe, where the drug received a positive recommendation from Europe’s Committee for Medicinal Products for Human Use. The company anticipates a decision from the European Commission by year-end, with the UK following suit through mutual recognition procedures. Eklund envisions the drug’s sales potential in Europe exceeding 150 million euros ($158 million) in five years.
In addition to their current achievements, Santhera and Catalyst are eagerly planning to secure new indications. They intend to collaborate on the cost of conducting clinical trials as they aim to expand the drug’s applications.
Agamree is administered as an oral suspension, with the dosage determined by the patient’s weight. The FDA’s approval encompasses patients aged 2 and older.
This landmark approval was based on data from a phase 2b trial, complemented by safety information gathered from three open-label studies spanning up to 48 months of treatment. Vamorolone demonstrated comparable efficacy to standard-of-care corticosteroids in a time-to-stand velocity test. The data also indicated a reduction in adverse events, along with improvements in bone health, growth trajectory, weight management, and behavior.
Given its orphan-drug and rare pediatric disease designations in DMD, Agamree will enjoy an additional seven years of exclusivity. Santhera anticipates patent protection in the U.S. until 2040 and in Europe until 2035. Vamorolone was initially developed by ReveraGen, a Maryland-based company specializing in DMD treatments. It was later acquired by Santhera after passing through the hands of Actelion, which eventually became Idorsia.
Following Thursday’s approval, Santhera received a $36 million milestone payment from Catalyst, with $10 million directed to Idorsia and $16 million to ReveraGen in accordance with prior licensing agreements.
DMD is the most prevalent form of muscular dystrophy, a genetic condition characterized by the inability to produce the essential dystrophin protein crucial for muscle function. This results in the loss of ambulatory ability and respiratory failure, with patients rarely living beyond 25 years. Approximately 70% of the 12,000 DMD patients in the U.S. are currently treated with steroids, according to Catalyst. While other DMD treatments exist, they are unlikely to supplant Agamree’s uptake. Santhera envisions a future where vamorolone is prescribed in combination with more advanced modalities, such as exon skippers or gene therapies, ultimately setting a new standard of care.
