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 infantile spasms will have access to Kigabeq® as quickly as possible,” adds Gilles ALBERICI, Chairman of ORPHELIA Pharma.

About Kigabeq®

Kigabeq® is the first paediatric formulation of vigabatrin, an essential anti-epileptic drug. Kigabeq® is available in 100 mg and 500 mg soluble and scored tablets for oral or (naso)gastric administration and is indicated for the first-line treatment of childhood spasms (West’s syndrome), a severe epileptic encephalopathy in infants. Kigabeq® has been developed exclusively for children and has been granted Paediatric Use Marketing Authorisation (PUMA).

About infantile spasms

West’s syndrome, or infantile spasms, is a severe epileptic encephalopathy of the infant that combines epileptic spasms, a psychomotor deterioration and an hypsarrhythmic electroencephalogram tracing. It is a rare disease, with an estimated incidence of 5 per 10,000 births. It can occur in infants with previously normal development or with a pre-existing delay; in all cases, infantile spasms have a strong impact on the psychomotor development. Pharmacological treatment must be started quickly to allow the spasms to stop and improve the prognosis.

 About the Ethypharm Group

Ethypharm is a European pharmaceutical company focused on two therapeutic areas: the Central Nervous System and Critical Care. Ethypharm markets its drugs directly in Europe and China, and with partners in North America and the Middle East where its drugs are in high demand. The Group employs more than 1,500 people, mainly in Europe and China.

Ethypharm works closely with authorities and healthcare professionals to ensure the appropriate use of and access to its medicines, by as many people as possible.

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.

Press contact

Avril PONNELLE / presse@ethypharm.com / + 33 (0)1 41 12 17 20

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

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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12 October 2020

ORPHELIA Pharma to organize a Symposium at the SIOP congress 2020
12 October 2020

Paris, October 12th, 2020 – ORPHELIA Pharma, a French biopharmaceutical company dedicated to the development and marketing of pediatric and orphan drugs announces today that it will organize a symposium at the Société Internationale d’Oncologie Pédiatrique (SIOP) congress 2020. The session will focus on the pediatric use of temozolomide in the treatment of relapsed or refractory pediatric tumors and will showcase the development of Kimozo a ready-to-use and taste-masked oral suspension of temozolomide adapted to children. “ORPHELIA Pharma is committed to improving the care of patients with pediatric and rare diseases, including developing innovative formulations to address children needs,” comments Jeremy Bastid, Chief Development Officer of ORPHELIA Pharma. “Our commitment to develop child-friendly medicines is a historical pillar of our company and this session at the SIOP meeting highlights our long-term close collaboration with the pediatric oncology community”. This industry symposium entitled “Improving children care with temozolomide oral suspension” will be held virtually on Friday, October 16th from 9:15 AM to 9:35 AM local time (Ottawa), from 3:15 PM. to 3:35 PM. Central European Time (CET) and accessible by connecting on the SIOP meeting website (SIOP-congress.eu) in the “Product theatre” section. Dr Lucas Moreno (Val d’Hebron Hospital, Spain) and Dr Dominique Valteau-Couanet (Gustave Roussy Hospital, France), two internationally renowned clinicians, will talk about the critical role of temozolomide in the management of relapsed or refractory pediatric tumors, including high-risk neuroblastomas. The third presenter, Dr Caroline Lemarchand, Chief Pharmaceutical Development Officer of ORPHELIA Pharma, will discuss the development of Kimozo, the first pediatric formulation of temozolomide under clinical development. We look forward to welcoming you during this unique event. About Kimozo Kimozo (also known as Ped-TMZ or ORP-005) is a ready-to-use and taste-masked oral suspension of temozolomide that is currently under clinical development to address children needs. Kimozo is an investigational medicinal product not yet approved for use or marketing anywhere in the world. About SIOP 2020 virtual congress The SIOP Annual Meetings bring together more than 2,500 leading clinicians and scientists from over 100 countries to share recent momentous scientific advances in the field of paediatric oncology. This congress represents an unique opportunity to stay up to date in this rapidly moving field and to bring the latest science to bedside. In light of the impact of the COVID-19 pandemic on travel restrictions and physical distancing requirements, the SIOP 2020 Annual Meeting will be held virtually on October 14-17, 2020.
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5 October 2020

Inclusion of the first patient in a clinical study evaluating Kimozo
5 October 2020

ORPHELIA announces the inclusion of the first patient in a clinical study evaluating Kimozo, its pediatric formulation of temozolomide Paris and Lyon, October 5th, 2020 – ORPHELIA Pharma, a French biopharmaceutical company dedicated to the development and marketing of pediatric and orphan drugs is pleased to announce the inclusion of the first-in patient in a clinical study aimed at demonstrating the bioequivalence between Kimozo, the first pediatric formulation of temozolomide under clinical development, and Temodal® capsule. Although not approved in this indication, temozolomide is an essential anticancer medicine recommended for use in treatment protocols of relapsed or refractory neuroblastoma, a condition that affects young children. However, the only oral formulations that are commercially available are in the form of capsules that are poorly adapted to children. Caretakers are thus compelled to open capsules and disperse the content into a soft food prior to administration to the child, which harbors several risks such as the uncertainty with regards to the delivered dose, the exposure of the caregiver to the cytotoxic drug and the instability of the drug in aqueous milieu. To overcome the risks this situation implies, Kimozo, a ready to use oral formulation has been specifically designed to address the needs of the pediatric population. Kimozo has been developed in collaboration with Gustave Roussy, one of the leading cancer centers in Europe. The current clinical trial conducted by ORPHELIA Pharma aims at demonstrating, first for regulatory purposes, the bioequivalence between Kimozo and the Temodal® capsules in adult patients having brain cancers, with additional clinical investigation to come in the pediatric population of interest. “We have reached an important milestone for Kimozo with the clinical trial approval from competent authorities and the recruitment of a first patient“, said Caroline Lemarchand, Chief Pharmaceutical Development Officer of ORPHELIA Pharma “We plan to enroll 30 patients by mid-2021 thanks to the support of the three neuro-oncology teams involved in the study: Professor Ducray of the Hospices Civils de Lyon (coordinating investigator), Professor Chinot of the Timone Hospital in Marseille and Dr Bronnimann of Saint-André Hospital in Bordeaux. ” “We are pleased to contribute to the development of this new pediatric formulation of temozolomide. A liquid form is unambiguously of interest for treating children.“, underlines Pr. François Ducray. Hugues Bienaymé, General Manager of ORPHELIA Pharma further comments: “This first administration is a major milestone in the development of Kimozo. We are now preparing the opening of our […]
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1 October 2020

Meet us at BIO-Europe 2020, October 26-29
1 October 2020

Meet us at BIO-Europe 2020, October 26-29 Jeremy Bastid, Chief Development Officer of ORPHELIA Pharma, will participate in the upcoming BIO-Europe conference to be held virtuallly on october 26-29, 2020. BIO-Europe is Europe’s largest partnering conference serving the global biotech industry. Jeremy will actively take part in this business event, registered under the name of the holding company, initiative OCTALFA.
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