ORPHELIA Pharma and Biocodex sign a license agreement for the distribution of Kigabeq® in 14 european countries

Paris and Gentilly – April 6th 2021 – ORPHELIA Pharma and Biocodex announce today the execution of an exclusive distribution and marketing agreement for Kigabeq® (vigabatrin) for most of the European Union territory, including France.

Kigabeq®, the first pediatric form of vigabatrin developed by ORPHELIA Pharma, is notably indicated in the treatment of infantile spasms (West syndrome). Kigabeq® is approved in Europe, where this medicine, intended exclusively for children, benefits from a centralized marketing authorization (Pediatric Use Marketing authorization, PUMA).

We are very pleased to release this collaboration agreement with Biocodex“, says Hugues BIENAYME, Founder and CEO of ORPHELIA Pharma, “With their long-standing experience in the field of pediatric epilepsy, Biocodex is certainly the best partner for Kigabeq® distribution in Europe“.

With Kigabeq®, the only pediatric presentation of vigabatrin, Biocodex is expanding its portfolio of drugs intended for rare and serious pathologies in children” adds Nicolas Coudurier, CEO of Biocodex, “ Kigabeq® and Diacomit® (stiripentol, developed and marketed by Biocodex) are two drugs which address unmet medical needs for young patients affected by severe and resistant to treatment epilepsies”.

Our objective is to make Kigabeq® available to all European clinicians, so that children affected by West syndrome can benefit.” concludes Gilles ALBERICI, President of ORPHELIA Pharma, “Thanks to this agreement with Biocodex, Kigabeq® will soon be prescribed in most of the European territory.”

About Kigabeq®

Kigabeq® is the first pediatric formulation of vigabatrin, an essential anti-epileptic drug. Presented as soluble and scored tablets of 100 mg and 500 mg for oral or nasogastric administration, Kigabeq® is notably indicated for the treatment of infantile spasms (West syndrome), an extremely serious epileptic encephalopathy of the infant. Kigabeq® was developed exclusively for children and has been granted European Pediatric Marketing Authorization (PUMA).

About infantile spasms

West syndrome, or infantile spasms, is an extremely serious epileptic encephalopathy in infants that combines epileptic spasms, psychomotor deterioration and a hypsarrhythmic type electroencephalogram. It is a rare disease, with an estimated incidence of 5 per 10,000 births. It can occur in an infant with previously normal development or with a pre-existing delay; in all cases, infantile spasms severely hamper the psychomotor development. Pharmacological treatment should be started quickly to allow the spasms to stop and to improve the prognosis.

 About Biocodex

Funded in 1953 beyond the development and marketing of the first probiotic strain in the world, Saccharomyces boulardii CNCMI-745, Biocodex from pioneer became one of leading international players within microbiota.

Biocodex further extended its activities to nervous central system diseases, with a focus on pediatric neurology, reinforcing its commitment to improve quality of life through discovery of innovative drugs addressed to unmet medical needs: full development of antiepileptic drug stiripentol (DIACOMIT®), indicated for the treatment of Dravet syndrome which is a serious and rare epilepsy observed in infants, has brought a valid therapeutic solution to neuropaediatricians.

Initially approved in Europe in 2007 then Canada and Japan in 2012, Diacomit® obtained Marketing Authorization in USA and Switzerland in 2018, Australia in 2019 and recently in Argentina. In numerous other countries, patients could access the drug through specific compassionate use programs. Dedicated medical team is in charge of orphan drugs portfolio, with permanent connexion with neuropaediatric specialists in UE and worldwide.

About ORPHELIA Pharma

ORPHELIA Pharma is a biopharmaceutical company based in Paris and Lyon that develops and markets drugs for the treatment of pediatric and orphan diseases.

Our mission is to provide patients with essential hospital products in the fields of epilepsy and oncology, with formulations that are suited to the pediatric population. Our first product, Kigabeq®, which was approved during the latter part of 2018 for the treatment of West Syndrome in particular, has been launched in several European countries. Our second product, Ivozall®, obtained a European marketing authorization at the end of 2019 for the treatment of acute lymphoblastic leukemia. ORPHELIA Pharma also conducts research projects through academic and industrial partnerships.

Contacts

Séverine MARTIN / orphelia@orphelia-pharma.eu / +33 (0)1 42 77 08 18

Véronique STALLER / v.staller@biocodex.fr /+33 (0)1 41 24 30 84

6 April 2021

License agreement for the distribution of Kigabeq® in 14 european countries
6 April 2021

ORPHELIA Pharma and Biocodex sign a license agreement for the distribution of Kigabeq® in 14 european countries Paris and Gentilly – April 6th 2021 – ORPHELIA Pharma and Biocodex announce today the execution of an exclusive distribution and marketing agreement for Kigabeq® (vigabatrin) for most of the European Union territory, including France. Kigabeq®, the first pediatric form of vigabatrin developed by ORPHELIA Pharma, is notably indicated in the treatment of infantile spasms (West syndrome). Kigabeq® is approved in Europe, where this medicine, intended exclusively for children, benefits from a centralized marketing authorization (Pediatric Use Marketing authorization, PUMA). “We are very pleased to release this collaboration agreement with Biocodex“, says Hugues BIENAYME, Founder and CEO of ORPHELIA Pharma, “With their long-standing experience in the field of pediatric epilepsy, Biocodex is certainly the best partner for Kigabeq® distribution in Europe“. “With Kigabeq®, the only pediatric presentation of vigabatrin, Biocodex is expanding its portfolio of drugs intended for rare and serious pathologies in children” adds Nicolas Coudurier, CEO of Biocodex, “ Kigabeq® and Diacomit® (stiripentol, developed and marketed by Biocodex) are two drugs which address unmet medical needs for young patients affected by severe and resistant to treatment epilepsies”. “Our objective is to make Kigabeq® available to all European clinicians, so that children affected by West syndrome can benefit.” concludes Gilles ALBERICI, President of ORPHELIA Pharma, “Thanks to this agreement with Biocodex, Kigabeq® will soon be prescribed in most of the European territory.” About Kigabeq® Kigabeq® is the first pediatric formulation of vigabatrin, an essential anti-epileptic drug. Presented as soluble and scored tablets of 100 mg and 500 mg for oral or nasogastric administration, Kigabeq® is notably indicated for the treatment of infantile spasms (West syndrome), an extremely serious epileptic encephalopathy of the infant. Kigabeq® was developed exclusively for children and has been granted European Pediatric Marketing Authorization (PUMA). About infantile spasms West syndrome, or infantile spasms, is an extremely serious epileptic encephalopathy in infants that combines epileptic spasms, psychomotor deterioration and a hypsarrhythmic type electroencephalogram. It is a rare disease, with an estimated incidence of 5 per 10,000 births. It can occur in an infant with previously normal development or with a pre-existing delay; in all cases, infantile spasms severely hamper the psychomotor development. Pharmacological treatment should be started quickly to allow the spasms to stop and to improve the prognosis.  About Biocodex Funded in 1953 beyond the development and marketing of […]
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2 February 2021

Opening of the operating pharmaceutical establishment
2 February 2021

ORPHELIA Pharma opens its operating pharmaceutical establishment (“laboratoire pharmaceutique exploitant”) in France Paris and Lyon, February 2nd, 2021. ORPHELIA Pharma, a French biopharmaceutical company dedicated to the development and marketing of pediatric and orphan drugs, announces today that it has opened its operating pharmaceutical establishment (“laboratoire pharmaceutique exploitant”) in France. According to the French Public Health Code (“Code de la Santé Publique”), ORPHELIA Pharma will now be able to commercialize medicines under its own name. The authorized activities include importation, sales, advertising, medical information, pharmacovigilance and batch monitoring. The establishment will also be able to launch and monitor early access programs (“Autorisation Temporaire d’Utilisation”, or ATU) on its own behalf or on behalf of third parties. “As of today, we will be able to commercialize our own medicines in France. We currently have two pediatric medicines which have been approved in Europe through a centralized procedure: Kigabeq® (vigabatrin) and Ivozall® (clofarabine)”, comments Laurent Martin, Chief Pharmaceutical Officer of ORPHELIA Pharma. “Becoming a pharmaceutical establishment will also allow us to initiate an early access program (ATU) for our experimental medicine Kimozo®, a liquid oral formulation of temozolomide developed for the treatment of relapsed or refractory neuroblastoma, following validation by health authorities”, he adds. “After having obtained the authorization to open our pharmaceutical establishment from the French medicines agency (ANSM) on November 18th, 2020, this effective opening is a major milestone for our company and is a testament to the efforts undertaken by the people at ORPHELIA Pharma”, concludes Hugues Bienaymé, General Manager. About ORPHELIA Pharma ORPHELIA Pharma is a biopharmaceutical company based in Paris and Lyon that develops and markets drugs for the treatment of paediatric and rare diseases. Our mission is to provide patients with essential hospital products in the fields of epilepsy and oncology with formulations adapted to the paediatric population. Our first product, Kigabeq®, received European marketing authorization in September 2018 and has been made available in several countries. Our second product, Ivozall®, obtained European marketing authorization in November 2019 for the treatment of acute lymphoblastic leukemia. ORPHELIA Pharma conducts research projects through academic and industrial collaborations.
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26 January 2021

Grant obtained from the region Île-de-France to support business activity development in Europe and the United States
26 January 2021

ORPHELIA Pharma awarded a grant from the region Île-de-France to support business activity development in Europe and in the US Paris and Lyon, January 26th, 2021. ORPHELIA Pharma, a French biopharmaceutical company dedicated to the development and marketing of paediatric and orphan drugs announces today that it has received the support from the region Île-de-France as part of the PM’up program to accelerate the development of the company in Europe and the US. ORPHELIA Pharma will receive a grant of 250 000 € aimed at supporting the international development of the company. “We are grateful to receive the support from region Île-de-France which will allow us to accelerate the development of our medicines portfolio worldwide. Our products address unmet medical needs of life-threatening paediatric diseases and our ambition is to make them available to all patients“, comments Jeremy Bastid, Chief Development Officer of ORPHELIA Pharma. “We will use the proceeds to implement the marketing operations of Kigabeq® in Europe and continue the efforts in finding partners for key territories elsewhere“, states Hugues Bienaymé, General Manager. “This program will also support the preparation of the US dossier and the evaluation of the market access strategy of Kimozo®, the first paediatric drinkable formulation of temozolomide currently under development for the treatment of relapsed or refractory neuroblastoma”, he adds. About PM’Up program The Région Ile-de-France supports the development of Small and Medium-sized Enterprises by providing financial assistance to support their growth strategy. PM’up is a grant of up to 250 000 € to finance a 3-year development plan. For more information, please visit https://www.iledefrance.fr/pmup. About ORPHELIA Pharma ORPHELIA Pharma is a biopharmaceutical company based in Paris and Lyon that develops and markets drugs for the treatment of paediatric and rare diseases. Our mission is to provide patients with essential hospital products in the fields of epilepsy and oncology with formulations adapted to the paediatric population. Our first product, Kigabeq®, received European marketing authorisation in September 2018 and is being launched in several countries. Our second product, Ivozall®, obtained European marketing authorisation in November 2019 for the treatment of acute lymphoblastic leukaemia. ORPHELIA Pharma conducts research projects through academic and industrial collaborations.
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19 January 2021

Signature of a licensing agreement for the development and marketing of Kigabeq® in China
19 January 2021

ORPHELIA Pharma and Ethypharm sign a licensing agreement for the development and marketing of Kigabeq® in China Saint-Cloud and Paris – January 19, 2021 – Ethypharm and ORPHELIA Pharma announce today the signature of an exclusive agreement for the development, registration and marketing in the People’s Republic of China of Kigabeq® (vigabatrin) indicated for the first-line treatment of infantile spasms (West syndrome).  Kigabeq®, the first paediatric form of vigabatrin developed by ORPHELIA Pharma, is approved and marketed in Europe, where this medicine, intended exclusively for children, has been granted a Paediatric Use Marketing Authorisation (PUMA). Kigabeq® is indicated for the first-line treatment of infantile spasms (West’s syndrome), a severe epileptic encephalopathy in infants. There is a significant unmet medical need in China for patients suffering from this rare and devastating form of epilepsy. By entering into this agreement, Ethypharm and ORPHELIA Pharma are committed to providing appropriate treatment for Chinese children suffering from this rare disease. As part of this agreement, ORPHELIA Pharma grants Ethypharm exclusive rights to Kigabeq® in the People’s Republic of China. After registration, Ethypharm will be responsible for the distribution and sale of Kigabeq® in China.  “Our objective is to make Kigabeq® available in many territories. After Europe and China, new agreements will be signed to enable the marketing of this formulation of vigabatrin, which is particularly suitable for children,” comments Hugues BIENAYME, founder and Managing Director of ORPHELIA Pharma.  “We are very pleased to establish this partnership with ORPHELIA Pharma. This collaboration brings an effective solution to young Chinese patients. The upcoming commercialisation of Kigabeq® is fully in line with our strategy to strengthen our rare disease franchise in China” said Bertrand DELUARD, President and CEO of Ethypharm.  Through this partnership, Ethypharm is strengthening its ambition to become a key player in the field of rare diseases in China. Operating successfully for more than 20 years in an ever-changing Chinese environment, Ethypharm has a fully integrated subsidiary located in Shanghai. “Our subsidiary is a partner of choice for companies wishing to make their existing internationally-marketed orphan drugs or drugs for rare diseases available to Chinese patients,” says François LIOT, General manager of Shanghai Ethypharm. “I am delighted to sign this agreement with Ethypharm, which is based on the value of Kigabeq® in paediatrics. Thanks to the in-depth knowledge that the Ethypharm Group is developing in China, we are very confident that chinese children suffering from […]
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