Company to Boost Orphan Drugs in Europe

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15 Nov. 2022. A start-up enterprise seeks to make drugs for treating rare diseases more readily available to physicians and patients in Europe.  Avanzanite Bioscience B.V., a company formed last year in Amsterdam, began operations today with what it claims is a business model designed to overcome current obstacles in Europe facing rare disease drugs.

Avanzanite Bio says the vast majority of new orphan drugs launched in Europe, those designed to treat for rare diseases, fail to reach their intended patients, nor do they achieve commercial or revenue goals. The company traces much of the problem to regulatory, distribution, and marketing variations in each European country, requiring more than 30 national strategies for getting these needed treatments in the hands of physicians, even after review and approval of new drugs by European authorities. As a result, says the company, many drug makers refuse to bring their orphan drugs to Europe or leave the market altogether.

To enable more European patients to benefit from orphan drugs, says Avanzanite Bio, requires a new business model. The company plans to partner with biotech and pharmaceutical companies, to gain exclusive licenses for distributing drugs that reach late clinical trial stages or after EU approval, to treat rare diseases. Avanzanite says it can offer orphan drug makers a single point of entry to European markets without building their own distribution or marketing infrastructures in the individual countries.

As an example, Avanzanite offers its partnership with SIFI S.p.A., a developer of eye disease therapies in Catania, Italy. SIFI produces treatments for infections and inflammation in the eye, including acanthamoeba keratitis, a rare infection affecting the cornea. While considered a rare disease, acanthamoeba keratitis is more likely to occur in people with soft contact lenses, where an amoeba, a single-cell microorganism, enters and damages tissue in the cornea. The disease can lead to eye pain, vision loss, and permanent blindness.

Managing complexities of product launches

SIFI created the drug Akantior, an anti-amoebic polymer as a treatment for acanthamoeba keratitis. In a conference paper last month, researchers from the company say nearly nine in 10 participants (87%) in a late-stage clinical trial receiving Akantior clinically resolved their infections, compared to 55 percent in real-world findings, while 62 percent of participants report full vision restored.

Even while based in Italy, SIFI says it employed Avanzanite Bio to help market the drug throughout Europe. Fabrizio Chines, SIFI’s chairman and CEO says in a statement that the Avanzanite team “demonstrated an impressive track record of managing the complexities of product launches across the fragmented European marketplace, particularly regarding pricing and reimbursement, physician engagement, and technical operations.”

Avanzanite Bio is founded by pharma and biotech entrepreneurs Adam Pilch and Anant Murthy. “We’ve cracked the code in deciphering this tricky landscape,” notes Pilch, “and now, with our initial strategic partnership with SIFI, we are ready to take our transformational model to the next level by bringing new medicines to patients with rare diseases.”

The company says it’s fully authorized to distribute medicinal products throughout the European Economic Area that includes European Union states, as well as Iceland, Liechtenstein, and Norway. Avanzanite does not disclose its investors or other financial details.

More from Science & Enterprise:

• Gene Therapy Stimulates Missing Enzymes in Rare Disease
• NIH Funds Decentralized Rare Disease Data Network
• FDA Issues Action Plan for Rare Neuro Diseases
• Micro Brain Robot Granted Orphan Status
• Trial OKed for Rare Disease Gene Therapy

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