Acidosis, Renal Tubular

CHMP opinion paves the way for Marketing Authorisation approval by the European Commission, currently anticipated in early August 2024. When approved, AKANTIOR will be the first licensed medicinal product for the treatment of acanthamoeba keratitis, a rare, severe eye infection that often results in blindness and eye loss. Phase III ODAK study demonstrated 87% of patients receiving AKANTIOR were cured within a median time of 4 months. AKANTIOR will be Avanzanite’s second commercialized orphan medicine, expanding the company’s pan-European footprint to 26 countries. AMSTERDAM--(BUSINESS WIRE)-- Avanzanite Bioscience B.V. (“Avanzanite” or the “Company”), a commercial-stage specialty pharmaceutical company, solely focused on medicines for rare diseases, announced today that the European Medicines Agency’s (“EMA”) Committee for Medicinal Products for Human Use (“CHMP”) issued a positive opinion recommending Marketing Authorisation of AKANTIOR® (polihexanide) for the treatment of acanthamoeba keratitis (“AK”) in adults and children from 12 years of age. This press release features multimedia. View the full release here: Adam Plich, Founder & CEO, Avanzanite Bioscience B.V. (Photo: Business Wire) Today’s announcement follows Avanzanite’s licensing and supply agreement with SIFI, a leading Italian ophthalmic healthcare company, that was announced in January 2023, granting Avanzanite the exclusive rights to commercialize AKANTIOR in 26 countries in the European Economic Area and Switzerland. Avanzanite’s Founder and CEO, Adam Plich, stated, “Avanzanite is a purpose-driven company whose sole mission is to ensure novel medicines reach patients suffering from rare diseases in every market across Europe and beyond. As such, the CHMP positive opinion takes us one step further in our unwavering pursuit of this important goal. No one facing such a monstrous disease should be left behind.” Mr. Plich added, “Through this strategic partnership, Avanzanite is unlocking access to a potential new treatment for those affected by AK in 26 European countries previously unavailable to SIFI.” This CHMP opinion represents the culmination of more than 15 years of research by SIFI based on a comprehensive data package demonstrating AKANTIOR’s quality, safety, and efficacy. In March 2024, results of the Orphan Drug for Acanthamoeba Keratitis (ODAK) pivotal 135-patient Phase III study were reported and published in Ophthalmology. The trial showed that 86,7% of patients receiving AKANTIOR were cured with a median time to cure of 4,1 months. Furthermore, 66,7% of patients treated with AKANTIOR achieved full vision restoration and only 7,5% required a cornea transplant. Following the CHMP’s recommendation to approve AKANTIOR for the treatment of AK, the European Commission (EC) will make its final Marketing Authorisation approval decision within approximately 2 months (currently expected in early August 2024). Meanwhile, AKANTIOR remains available through a pre-authorisation distribution program which, to date, has facilitated treatment access to more than 180 patients across 12 European countries. Following the successful commercialization of Sibnayal®, AKANTIOR will be the second rare disease medicine commercialized by Avanzanite, expanding the company’s footprint across 26 European countries. In February 2023, Avanzanite announced its exclusive distribution agreement with Paris-based specialty pharmaceutical company Advicenne for Sibnayal, approved in the European Union for the treatment of children and adults with distal renal tubular acidosis (dRTA), a rare genetic or acquired kidney disease. Avanzanite’s dedicated team supplies Sibnayal across 8 countries. ABOUT AVANZANITE BIOSCIENCE: Founded in 2022, Avanzanite is a privately-funded, commercial-stage, specialty pharmaceutical company solely focused on medicines for rare diseases. At Avanzanite we believe all patients should benefit from novel orphan or niche medicines regardless of where they live. Our vision is that no one is left behind when facing a debilitating rare disease. We also believe the people who have dedicated their careers to the research & development of novel orphan or niche medicines deserve to witness their tremendous efforts impacting patients’ lives – the work of these heroes should not lie in vain. We make this happen by acquiring, licensing, or distributing approved or late-stage medicines for rare diseases and commercializing them even where nobody else will. Headquartered in Amsterdam in the Netherlands, Avanzanite has commercial infrastructure and technical operations across Europe. Please visit for additional information. ABOUT ACANTHAMOEBA KERATITIS (AK): AK is a rare, severe, and progressive parasitic corneal infection caused by Acanthamoeba, a free-living amoeba. Urgent medical intervention is required as the disease often leads to blindness and eye loss. AK often requires single or multiple corneal transplant procedures with comparably low graft survival rates. Patients report unbearable pain and extreme light sensitivity and can rarely work or lead normal lives until symptoms resolve, leaving patients with trauma for life. While still very rare, the incidence of AK has grown rapidly in recent years. AK primarily affects contact lens wearers, accounting for 50% of blindness among them. ABOUT AKANTIOR®: AKANTIOR (polihexanide) is an anti-amoebic polymer that has been granted Orphan Drug Designation (ODD) by both the European Medicines Agency ("EMA") and Food and Drug Administration ("FDA") for AK. It acts on both the trophozoites and cysts of the protozoan Acanthamoeba. It is formulated at a unique high 0.8 mg/ml dose strength which makes it possible to administer through a validated and standardized, day-only protocol as monotherapy eye drops in single-dose containers. Polihexanide is also being developed by SIFI for the treatment of fungal keratitis for which indication it also has FDA ODD. View source version on businesswire.com: Contacts Adam Plich, Founder & CEO Avanzanite Bioscience B.V. +31 20 301 21 13 media@avanzanite.com Source: Avanzanite Bioscience B.V. Smart Multimedia Gallery Logo Video My journey with Acanthamoeba Keratitis, Juliette Vila Sinclair Spence, Founder of the Acanthamoeba Keratitis Eye Foundation. Photo Adam Plich, Founder & CEO, Avanzanite Bioscience B.V. (Photo: Business Wire) View this news release and multimedia online at:

Akantior’s commercialisation rights were acquired by Netherlands-based Avanzanite in January this year. Image credit: Shutterstock / Kateryna Kon. The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has backed approval for Akantior (polihexanide), a potential first medicine to treat acanthamoeba keratitis. Recommended Buyer's Guides Buyer's Guide Leading Guide to Packaging Materials, Containers and Containment Services for the Pharmaceutical Industry Buyer's Guide Leading Guide to Clinical Packaging Companies in contract Manufacturing for the Pharmaceutical Industry The CHMP will now send their recommendation of marketing authorisation – for patients aged 12 years and older with the infection – to the European Commission, with a decision expected in August 2024, according to a 3 June press release. Developed by Italian ophthalmic healthcare company SIFI , Akantior’s commercialisation rights were acquired by Netherlands-based Avanzanite in January this year. The licensing and supply agreement includes 26 countries in the European Economic Area and Switzerland. Acanthamoeba keratitis is an eye infection caused by an Acanthamoeba amoeba. The microorganism infects the cornea, leading to possible vision loss and blindness. The infection, which usually affects contact lens wearers, is treated off-label via various combinations of antiseptic and antifungal agents and, according to Avanzanite, by conducting corneal transplants. Akantior is an anti-amoebic polymer that works on both forms of Acanthamoeba in the eye – the mobile trophozoites and the infective cysts. The drug is administered as monotherapy eye drops at 0.8mg/ml dose. Akantior’s active substance is polyhexanide. See Also: The tiny pills that may send shockwaves across immunology Roche’s PI3K inhibitor secures breakthrough status in breast cancer The positive CHMP opinion was based on more than 15 years of data, Avanzanite said. This includes a Phase III trial involving 135 patients with the infection. Results from the trial (NCT03274895) showed that 86.7% of participants receiving the drug were cured with a median time to cure of 4.1 months. Only 7.5% of patients required a cornea transplant, with 66.7% regaining full vision after treatment with Akantior. Avanzanite’s CEO Adam Plich said the positive CHMP opinion takes the company “one step closer” to delivering the medicine to patients with the rare disease. Akantior has been awarded orphan drug status by both the EMA and the US Food and Drug Administration (FDA) – meaning the company has enjoyed a suite of pre-market benefits and could be eligible for market exclusivity in the regions if approved. In February 2023, Avanzanite announced an exclusive distribution agreement with French pharma company Advicenne for distal renal tubular acidosis (dRTA) treatment Sibnayal (potassium hydrogen carbonate / potassium citrate).

mAppointment of Marc-Andre Goldschmidt as Country Manager of Germany fortifies broader corporate initiative of strategically investing in human capital Goldschmidt brings a successful track record of multiple product launches of innovative medicines for the treatment of rare diseases in Germany Goldschmidt will initially focus on the growth of Avanzanite’s pediatric nephrology franchise AMSTERDAM--(BUSINESS WIRE)-- Avanzanite Bioscience B.V. (“Avanzanite” or the “Company”), a specialty commercial-stage pharmaceutical company with a corporate mission of boosting orphan drug access and unlocking their full potential in European markets, today announced the appointment of Marc-Andre Goldschmidt as Country Manager of Germany. This press release features multimedia. View the full release here: Marc-Andre Goldschmidt (Photo: Business Wire) “We’re excited to enlist Marc-Andre with this critical post at Avanzanite, adding to our high-caliber talent team to drive strong performance across our organization going forward,” stated Avanzanite’s Founder and CEO, Adam Plich. “With our growing momentum and continued business execution, it’s more important than ever to strategically add to our talent pool and position ourselves for the highest level of success in product sales across Germany and Europe.” Mr. Plich continued, “We brought Marc-Andre on board to spearhead these efforts, as he demonstrated this level of robust performance consistently throughout his career. In the near term, Marc-Andre will be laser-focused on driving the Company’s product launch of a commercially-ready treatment for distal renal tubular acidosis (dRTA), a rare kidney disease leading to stunted growth in children, brittle bones or rickets, kidney stones and life-threatening hypokalemia. We are confident his role will expand over time as Avanzanite continues to add new products to its portfolio of orphan drugs,” concluded Mr. Plich. Prior to Avanzanite, Mr. Goldschmidt served most prominently as national sales manager of neurology for Alexion Pharmaceuticals, where he filled senior sales positions with growing responsibility for six years. At Alexion, Mr. Goldschmidt was instrumental in accomplishing 220% growth in product sales of select orphan drugs during the COVID-19 pandemic, amongst other accomplishments. Immediately prior to Avanzanite, Mr. Goldschmidt served as business unit director at Stada, where he led the team that achieved 75% market share growth in the neurology therapy area. Earlier in his career, Mr. Goldschmidt was a district sales manager at Amgen with consistent and successful results. “Together with my colleagues in Germany and Europe, we have a great opportunity and mission to significantly improve the lives of people with rare diseases,” stated Mr. Goldschmidt. “Because of this purpose-driven mission, I’m enthusiastic to join Avanzanite and look forward to giving my all every single day and deepening our reach in pediatric nephrology and beyond throughout Germany.” ABOUT AVANZANITE BIOSCIENCE: Derived from the word ‘Tanzanite’ – one of the rarest and most underappreciated gemstones on earth – Avanzanite was formed to capitalize on two primary objectives, one economic, and one humanitarian. The economic goal is to offer end-to-end commercialization and distribution partnership with research-based biopharmaceutical originators to unlock the full value of their orphan medicines in European markets; this is the unique expertise and core competency of the Avanzanite team through its decades of operating experience navigating this area. Equally important, the Company's humanitarian goal stems from our commitment to enabling access to novel medicines for patients who suffer from orphan diseases regardless of where in Europe they live. Go to for additional information. View source version on businesswire.com: Contacts Avanzanite Bioscience B.V. +31 20 301 21 13 +49 800 0002544 investors@avanzanite.com media@avanzanite.com Source: Avanzanite Bioscience B.V. Smart Multimedia Gallery Photo Marc-Andre Goldschmidt (Photo: Business Wire) Logo View this news release and multimedia online at: