Egetis has secured a green light in Europe for its ultra rare disease drug Emcitate and the Swedish biotech is looking to hit the ground running with a first launch in the next few months on its own.
The European Commission has granted approval of Emcitate (tiratricol) for the treatment of patients with monocarboxylate transporter 8 (MCT8) deficiency. Also known as Allan-Herndon-Dudley syndrome, the rare congenital disorder is characterized by increased serum levels of thyroid hormone and until now, no therapy has been available.
The approval, which comes two months after a positive recommendation from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency, is primarily based on data from an investigator-initiated Phase II study called Triac I. That data showed that Emcitate reduced levels of the thyroid hormone T3 in the serum by more than 63% at month 12 and all patients (n=40) who completed the trial improved in at least one of the study endpoints: body weight, resting heart rate or systolic blood pressure.
CEO Nicklas Westerholm said that the approval of Emcitate as the first treatment for patients with MCT8 deficiency “represents the single most important milestone in Egetis’ history and a major step forward in building a sustainable rare disease company.” He added that the company would look forward to initiating pricing and reimbursement processes and discussions in Europe and expect the first launch in the second quarter of 2025.
Egetis, which was formed at the end of 2020 through the merger of PledPharma and Rare Thyroid Therapeutics and has been mentioned as a takeover target, is going it alone for launches in Europe. On the firm’s Q3 call, Westerholm said that “the go-to-market strategy is very simple” as like most companies, the Stockholm-based biotech will initially focus on Germany, where “there’s free pricing through commercialization during the discussion of reimbursement for up to 12 months.”
Egetis will then focus on Italy, France and Spain and while the reimbursement discussions “will take time, roughly one to two years,” he said that the company expected to get “orphan premium price” in Europe. At an investor day in December following the CHMP recommendation, Westerholm noted that “we’ve invested more than €90m since the back end of 2020 to make this happen” and getting approval “in the context of being a small Swedish biotech with only 40 employees” was quite an achievement.
US Trial Enrolment Challenge
Egetis has also got its sights set on selling Emcitate itself in the US but a filing across the Atlantic is dependent on the successful outcome of the ReTRIACt trial, which is still recruiting. After dialogue with the US Food and Drug Administration, Egetis is conducting a study in at least 16 evaluable patients to verify the results on T3 levels seen in previous studies and recruitment has been a challenge. Getting patients that fit the inclusion criteria has been harder than the company had expected and enrolment is further hampered by the distances patients and their caregivers have to travel to get to trial sites.
If Emcitate gets approved in the US, Egetis would receive a priority review voucher which could be sold for around $150m at the going rate. This would provide a healthy boost to the cash position of the company which raised SEK300m (approximately $30m) through an oversubscribed share issue in September led by US healthcare investor Frazier Life Sciences.
In November 2023, a licensing deal for Emcitate in Japan was inked with Fujimoto Pharmaceutical Corp which is responsible for all regulatory interactions with the country’s Pharmaceuticals and Medical Devices Agency (PMDA). The ministry of health in Japan issued new guidelines for rare diseases in October this year allowing submissions without generating any clinical data from the country and this could lead to an earlier-than-expected approval there.
Egetis also sees the Middle East and North Africa, and Turkey as promising markets for Emcitate. It is looking at local collaborations for those territories and several discussions with potential partners have taken place.
The company is also contemplating a Phase II study of Emcitate in collaboration with academia in patients with resistance to thyroid hormone beta. It is a rare genetic disorder with no approved treatment that affects 1-2 individuals per 40,000 live births.
