Rare epilepsies as a whole account for 20-30% of epilepsies, but knowledge about prognostic factors is currently limited. This means that it is difficult to provide adequate information to families at diagnosis and during follow-up. Prognostic factors are also important for management as they can have an impact on the patient's outcome (time to intervention, choice of one molecule over another, etc.). Finally, few treatments are currently available for these epilepsies. One of the limitations to the development of treatments is the lack of real life data as it is difficult to create reliable primary endpoints such as the rate of patients becoming seizure free naturally compared to a therapeutic intervention.
The aim of this real-life study is to evaluate the response to treatment as well as to see the evolution of cognitive and psychiatric comorbidities. As explained above, there are very few randomised trials except for 3 rare epilepsies (infantile spasm syndrome, Dravet syndrome, Lennox-Gastaut syndrome). This has led to the virtual absence of management recommendations, including for the three syndromes mentioned above, where attempts at treatment algorithms have been proposed, although these have not been able to be considered as evidence-based recommendations.
As a result, there is some diversity in the management of rare epilepsies from one centre to another. However, this diversity in management can be an asset in a real-life study. This will make it possible to compare different management methods, both in terms of seizure control and medium-term outcome.

开始日期2021-12-01

申办/合作机构

Assistance Publique des Hôpitaux de Paris SA

[+1]

EUCTR2020-003733-38-DE / Not yet recruiting临床1期

TEMOkids study (ORP-TMZ-I- b): A Population pharmacokinetic, acceptability and safety study for KIMOZO, a paediatric oral suspension of temozolomide - TEMOkids study

开始日期2021-07-16

申办/合作机构

ORPHELIA Pharma SAS

NCT04610736 / Active, not recruiting临床1期

TEMOkids Study: A Population Pharmacokinetic, Acceptability and Safety Study for KIMOZO, a Paediatric Oral Suspension of Temozolomide

Non-randomized, international, multi-centre, open-label, single arm study to determine the pharmacokinetic (PK) parameters of a single dose of an oral suspension of temozolomide (KIMOZO) in the pediatric population aged 1 year and over.

开始日期2021-03-16

申办/合作机构

ORPHELIA Pharma SAS

[+1]

100 项与 ORPHELIA Pharma SAS 相关的临床结果

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0 项与 ORPHELIA Pharma SAS 相关的专利（医药）

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项与 ORPHELIA Pharma SAS 相关的文献（医药）

2023-01-01·Therapies

Drug repurposing: From the discovery of a useful pharmacological effect to making the treatment available to the patient

Article

作者: Bernasconi, Antoine ; Deplanque, Dominique ; Cougoule, Céline ; Beghyn, Terence ; Del Bano, Joanie ; Bernard, Claude ; Fetro, Christine ; Demiot, Claire ; Lechat, Philippe ; Ferry, Antoine ; Bienayme, Hugues ; Lebrun-Vignes, Bénédicte

The repurposing of a medicine already on the market to a new indication could be an opportunity to respond rapidly to a therapeutic need not yet covered, particularly in the context of rare and neglected diseases, or health emergencies. However, at each stage, difficulties may arise that will prevent the repurposed drug from being provided to patients. Beyond fortuity or a systematic strategy to detect a useful pharmacological effect, the implementation of the preclinical and clinical stages is sometimes complicated by the difficulty of accessing the molecule and its pharmaceutical data. Furthermore, relevant clinical results will not always be sufficient to ensure that a marketing authorisation is obtained or that patients receive satisfactory care. In addition to describing these various obstacles, the round table provided an opportunity to put forward recommendations for overcoming them, in particular the creation of a public-private partnership structure with sufficient funding to be able to offer individualised support for projects up to and including the marketing application.

2023-01-01·Therapies

Repositionnement des médicaments : de la découverte d’un effet pharmacologique utile à la mise à disposition du traitement pour le patient

Article

作者: Deplanque, Dominique ; Bernasconi, Antoine ; Cougoule, Céline ; Beghyn, Terence ; Del Bano, Joanie ; Bernard, Claude ; Fetro, Christine ; Demiot, Claire ; Lechat, Philippe ; Ferry, Antoine ; Bienayme, Hugues ; Lebrun-Vignes, Bénédicte

2022-06-01·Journal of Clinical Oncology

A bioequivalence study of a novel liquid and ready-to-use temozolomide oral suspension and temozolomide capsules in patients with primary tumors central nervous system malignancies.

作者: Ramirez, Carole ; Bastid, Jeremy ; Chinot, Olivier L. ; Durando, Xavier ; Bronnimann, Charlotte ; Ducray, Francois ; Maxime, Fontanilles ; Bienaymé, Hugues ; Estrade, Florian ; Lemarchand, Caroline ; Robert, Marie

e22008 Background: Oral temozolomide capsule is approved for the treatment of glioma and malignant glioblastoma in adults and in Europe in children over 3-years. As recommended by international pediatric medical associations, temozolomide is also used for the treatment of high-risk relapsed or refractory neuroblastoma, a solid tumor affecting young children. Nevertheless, capsules are not adapted to the pediatric population leading caregivers to handle temozolomide capsules, which bears major risks (i.e. dose inaccuracy, temozolomide instability and exposure to cytotoxic drug). To overcome this situation, a temozolomide oral suspension (Kimozo) was developed. The aim of this phase I study was to demonstrate bioequivalence between the temozolomide oral suspension and the temozolomide capsules (Temodal) and to assess the general and local safety in adult patients. Methods: A randomized, open-label, two-way crossover, single-dose bioequivalence study was performed in 8 centers. Adult patients with primary malignancies and treated with temozolomide 200 mg/m² as monotherapy received a single oral administration of the oral suspension (test) or capsule (reference) on days 1 and 2 of a 5-day cycle, depending on the randomization, and under fasting conditions. Fourteen blood samples were collected over 10-hr in each period for pharmacokinetic purpose. General and buccal safety was assessed along the study. The assessment of bioequivalence was based upon 90% confidence intervals (CI) for the ratio of the population geometric means (test/reference) for maximum plasma concentration (Cmax) and area under the curve (AUC0-t). Results: Among the thirty-six patients enrolled in the study, thirty were assessable for pharmacokinetic primary endpoint. The point estimate and the 90% CI of the ratios of Cmax and AUC0-t were 107.62 (98.07-118.09) and 97.18 (95.05-99.35), respectively. The results obtained satisfy the bioequivalence criteria of the Bioequivalence Guidelines (90% CI between 80.00% and 125.00%). Neither serious adverse events nor adverse events of special interest (i.e. mucositis) were reported. Conclusions: The oral suspension of temozolomide (Kimozo) and capsule of temozolomide (Temodal) are bioequivalent under fasting conditions in patients with CNS primary malignancies, supporting that they are therapeutic equivalent. Clinical trial information: NCT04467346.

项与 ORPHELIA Pharma SAS 相关的新闻（医药）

2024-10-03

·Pharmaceutical Technology

A rational design approach to RNA targeting could replace large-scale screening

Maria Duca speaking at the ELRIG Drug Discovery conference in London, UK. Credit: Frankie Fattorini/ GlobalData As interest mounts in using RNA as a therapeutic target, a group at the Institute of Chemistry in Nice is overcoming the need for lengthy, expensive screening of candidate compounds with a rational design approach. Dr. Maria Duca, head of the Targeting of Nucleic Acids team, outlined her team’s novel approach to RNA-targeting drug discovery, in a presentation today (3 October) at the ELRIG Drug Discovery 2024 conference in London, UK. Duca pointed out, “If we look at currently marketed drugs, these mainly target proteins, but these proteins represent a very small percentage of what the human genome encodes. If we take into account non-coding RNAs as potential targets for drugs, we could really widen the landscape of current medicinal chemistry.” However, according to Duca, “The problem is that you don’t have many rational design methodologies in order to design your compound specific for a particular RNA target. So, everything is mostly based on screenings.” Instead of mass screenings, Duca and her team assemble and test novel molecular conjugates to be target-specific and therapeutically effective. Duca said some of the more promising candidates created using this approach have potential in oncological and infectious disease indications. See Also: Advancing Pharmaceutical Security: The Role of SECURtracers in On-Dose Authentication Healthcare Payers for the Pharmaceutical Industry Duca outlined the discovery process behind her team’s conjugate against microRNA-327 (miRNA-327), an oncogenic RNA implicated in gastric cancer. The team conjugated the aminoglycoside neomycin with different artificial nuclear bases. The conjugates produced were screened in vitro for their selectivity and inhibitive activity towards miRNA-327. Promising candidates from these screens could then be studied in vivo. In gastrinoma cancer cells, which are characterised by miRNA-327 overexpression, the team found the chosen candidates inhibited cell proliferation in a dose-dependent manner. Imaging the conjugate-RNA interactions allowed them to further refine these neomycin conjugates for their miRNA-327 affinity and inhibition. Through this same process of design, in vitro screening, in vivo testing, and further structural refinement of conjugates, Duca and her team have produced therapeutic candidates targeting other RNAs. For example, they built another library of candidates targeted against miRNA-21 which is overexpressed in glioblastoma, a type of brain cancer. Applying the same rational design process, researchers produced candidates that inhibited glioblastoma cell proliferation, in a way that was comparable with the efficacy of temozolomide, one of the few palliative treatments for glioblastoma, marketed as Kimozo / Kizfizo by Orphelia Pharma. mRNA vaccine coverage on Pharmaceutical Technology (Or Clinical Trials Arena) is supported by Trilink . Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content. Free Whitepaper mRNA capping: low cost, high reward Are you aware of the latest mRNA capping techniques? The success of mRNA-based Covid vaccines has seen the technology boom in popularity – but capping is an essential part of the manufacturing process, and pharmaceutical firms face a range of choices on how to do it. This free whitepaper assesses differences in price, time, complexity and availability for the methods on offer – and provides essential insights on TriLink’s trailblazing CleanCap® approach. Fill in your details to find out more. Thank you. You will receive an email shortly. Please check your inbox to download the Whitepaper. By Trilink Thematic By downloading this Whitepaper, you acknowledge that GlobalData may share your information with Trilink Thematic and that your personal data will be used as described in their Privacy Policy

疫苗信使RNAsiRNA

2023-04-20

·Pharmaceutical Technology

ORPHELIA Pharma chooses Tanner Pharma to distribute Kimozo

Kimozo 40mg/ml is a drinkable formulation of temozolomide, approved to treat certain brain tumours in Europe. Credit: Nephron / commons.wikimedia.org. French biopharmaceutical company ORPHELIA Pharma has entered an exclusive agreement with Tanner Pharma Group to manage the supply and distribution of Ped-TMZ, also known as Kimozo (temozolomide 40mg/ml, oral suspension) in European countries. Free Buyers Guide Leading Guide to Pharmaceutical Logistics and Pharma Supply Chain Companies for the Pharmaceutical Industry The document includes detailed information on the manufacturers and suppliers and their products, along with contact details, to inform your purchasing decision. 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British (+1284) Virgin Islands - US (+1340) Wallis & Futuna (+681) Yemen (North)(+969) Yemen (South)(+967) Zambia (+260) Zimbabwe (+263) Country UK USA Afghanistan Åland Islands Albania Algeria American Samoa Andorra Angola Anguilla Antarctica Antigua and Barbuda Argentina Armenia Aruba Australia Austria Azerbaijan Bahamas Bahrain Bangladesh Barbados Belarus Belgium Belize Benin Bermuda Bhutan Bolivia Bonaire, Sint Eustatius and Saba Bosnia and Herzegovina Botswana Bouvet Island Brazil British Indian Ocean Territory Brunei Darussalam Bulgaria Burkina Faso Burundi Cambodia Cameroon Canada Cape Verde Cayman Islands Central African Republic Chad Chile China Christmas Island Cocos Islands Colombia Comoros Congo Democratic Republic of the Congo Cook Islands Costa Rica Côte d"Ivoire Croatia Cuba Curaçao Cyprus Czech Republic Denmark Djibouti Dominica Dominican Republic Ecuador Egypt El Salvador Equatorial Guinea Eritrea Estonia Ethiopia Falkland Islands Faroe Islands Fiji Finland France French Guiana French Polynesia French Southern Territories Gabon Gambia Georgia Germany Ghana Gibraltar Greece Greenland Grenada Guadeloupe Guam Guatemala Guernsey Guinea Guinea-Bissau Guyana Haiti Heard Island and McDonald Islands Holy See Honduras Hong Kong Hungary Iceland India Indonesia Iran Iraq Ireland Isle of Man Israel Italy Jamaica Japan Jersey Jordan Kazakhstan Kenya Kiribati North Korea South Korea Kuwait Kyrgyzstan Lao Latvia Lebanon Lesotho Liberia Libyan Arab Jamahiriya Liechtenstein Lithuania Luxembourg Macao Macedonia, The Former Yugoslav Republic of Madagascar Malawi Malaysia Maldives Mali Malta Marshall Islands Martinique Mauritania Mauritius Mayotte Mexico Micronesia Moldova Monaco Mongolia Montenegro Montserrat Morocco Mozambique Myanmar Namibia Nauru Nepal Netherlands New Caledonia New Zealand Nicaragua Niger Nigeria Niue Norfolk Island Northern Mariana Islands Norway Oman Pakistan Palau Palestinian Territory Panama Papua New Guinea Paraguay Peru Philippines Pitcairn Poland Portugal Puerto Rico Qatar Réunion Romania Russian Federation Rwanda Saint Helena, Ascension and Tristan da Cunha Saint Kitts and Nevis Saint Lucia Saint Pierre and Miquelon Saint Vincent and The Grenadines Samoa San Marino Sao Tome and Principe Saudi Arabia Senegal Serbia Seychelles Sierra Leone Singapore Slovakia Slovenia Solomon Islands Somalia South Africa South Georgia and The South Sandwich Islands Spain Sri Lanka Sudan Suriname Svalbard and Jan Mayen Swaziland Sweden Switzerland Syrian Arab Republic Taiwan Tajikistan Tanzania Thailand Timor-Leste Togo Tokelau Tonga Trinidad and Tobago Tunisia Turkey Turkmenistan Turks and Caicos Islands Tuvalu Uganda Ukraine United Arab Emirates US Minor Outlying Islands Uruguay Uzbekistan Vanuatu Venezuela Vietnam British Virgin Islands US Virgin Islands Wallis and Futuna Western Sahara Yemen Zambia Zimbabwe Kosovo By downloading this case study, you acknowledge that GlobalData may share your information with Pharma Buyers Guide and that your personal data will be used as described in their Privacy Policy Submit Visit our Privacy Policy for more information about our services, how GlobalData may use, process and share your personal data, including information on your rights in respect of your personal data and how you can unsubscribe from future marketing communications. Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address. Thank you. Please check your email to download the Buyers Guide. Kimozo 40mg/ml is a liquid, taste-masked and ready-to-use drinkable formulation of an anti-cancer drug, temozolomide, approved for use in Europe as capsules or as an IV formulation to treat certain brain tumours. It was developed by a partnership between ORPHELIA Pharma’s development team and pharmacists and clinicians of Gustave Roussy Hospital. Tanner Pharma will be responsible for providing access to Kimozo in European countries through a named patient programme, allowing doctors to prescribe investigational or approved drugs to patients before their commercial availability. The programme will be conducted chiefly in countries where Kimozo is already used as investigational medication in the TEMOkids clinical trial, backed by ORPHELIA Pharma. Tanner Pharma will also be responsible for managing all aspects of supply including logistics, national regulatory oversight, healthcare practitioner enquiry management and liaison with national authorities. The deal covers all European territories except France. ORPHELIA Pharma general manager Hugues Bienaymé stated: “Kimozo is currently available in France through an early access programme, and ORPHELIA has already received requests from physicians from other countries. “To meet these demands, ORPHELIA Pharma will use the services of Tanner Pharma, a world-leading pharmaceutical services provider with extensive experience in enabling access to innovative therapies when no other treatment options exist. “With this agreement, our ambition is to make Kimozo available to children in need of a ready-to-use drinkable suspension of temozolomide, anywhere in Europe and with no delay, in strict adherence with local regulations.” Free Buyers Guide Leading Guide to Pharmaceutical Logistics and Pharma Supply Chain Companies for the Pharmaceutical Industry The document includes detailed information on the manufacturers and suppliers and their products, along with contact details, to inform your purchasing decision. By Pharma Buyers Guide Submit Country Code UK (+44) USA (+1) Algeria (+213) Andorra (+376) Angola (+244) Anguilla (+1264) Antigua & Barbuda (+1268) Argentina (+54) Armenia (+374) Aruba (+297) Australia (+61) Austria (+43) Azerbaijan (+994) Bahamas (+1242) Bahrain (+973) Bangladesh (+880) Barbados (+1246) Belarus (+375) Belgium (+32) Belize (+501) Benin (+229) Bermuda (+1441) Bhutan (+975) Bolivia (+591) Bosnia Herzegovina (+387) Botswana (+267) Brazil (+55) Brunei (+673) Bulgaria (+359) Burkina Faso (+226) Burundi (+257) Cambodia (+855) Cameroon (+237) Canada (+1) Cape Verde Islands (+238) Cayman Islands (+1345) Central African Republic (+236) Chile (+56) China (+86) Colombia (+57) Comoros (+269) Congo (+242) Cook Islands (+682) Costa Rica (+506) Croatia (+385) Cuba (+53) Cyprus North (+90392) Cyprus South (+357) Czech Republic (+42) Denmark (+45) Djibouti (+253) Dominica (+1809) Dominican Republic (+1809) Ecuador (+593) Egypt (+20) El Salvador (+503) Equatorial Guinea (+240) Eritrea (+291) Estonia (+372) Ethiopia (+251) Falkland Islands (+500) Faroe Islands (+298) Fiji (+679) Finland (+358) France (+33) French Guiana (+594) French Polynesia (+689) Gabon (+241) Gambia (+220) Georgia (+7880) Germany (+49) Ghana (+233) Gibraltar (+350) Greece (+30) Greenland (+299) Grenada (+1473) Guadeloupe (+590) Guam (+671) Guatemala (+502) Guinea (+224) Guinea - 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British (+1284) Virgin Islands - US (+1340) Wallis & Futuna (+681) Yemen (North)(+969) Yemen (South)(+967) Zambia (+260) Zimbabwe (+263) Country UK USA Afghanistan Åland Islands Albania Algeria American Samoa Andorra Angola Anguilla Antarctica Antigua and Barbuda Argentina Armenia Aruba Australia Austria Azerbaijan Bahamas Bahrain Bangladesh Barbados Belarus Belgium Belize Benin Bermuda Bhutan Bolivia Bonaire, Sint Eustatius and Saba Bosnia and Herzegovina Botswana Bouvet Island Brazil British Indian Ocean Territory Brunei Darussalam Bulgaria Burkina Faso Burundi Cambodia Cameroon Canada Cape Verde Cayman Islands Central African Republic Chad Chile China Christmas Island Cocos Islands Colombia Comoros Congo Democratic Republic of the Congo Cook Islands Costa Rica Côte d"Ivoire Croatia Cuba Curaçao Cyprus Czech Republic Denmark Djibouti Dominica Dominican Republic Ecuador Egypt El Salvador Equatorial Guinea Eritrea Estonia Ethiopia Falkland Islands Faroe Islands Fiji Finland France French Guiana French Polynesia French Southern Territories Gabon Gambia Georgia Germany Ghana Gibraltar Greece Greenland Grenada Guadeloupe Guam Guatemala Guernsey Guinea Guinea-Bissau Guyana Haiti Heard Island and McDonald Islands Holy See Honduras Hong Kong Hungary Iceland India Indonesia Iran Iraq Ireland Isle of Man Israel Italy Jamaica Japan Jersey Jordan Kazakhstan Kenya Kiribati North Korea South Korea Kuwait Kyrgyzstan Lao Latvia Lebanon Lesotho Liberia Libyan Arab Jamahiriya Liechtenstein Lithuania Luxembourg Macao Macedonia, The Former Yugoslav Republic of Madagascar Malawi Malaysia Maldives Mali Malta Marshall Islands Martinique Mauritania Mauritius Mayotte Mexico Micronesia Moldova Monaco Mongolia Montenegro Montserrat Morocco Mozambique Myanmar Namibia Nauru Nepal Netherlands New Caledonia New Zealand Nicaragua Niger Nigeria Niue Norfolk Island Northern Mariana Islands Norway Oman Pakistan Palau Palestinian Territory Panama Papua New Guinea Paraguay Peru Philippines Pitcairn Poland Portugal Puerto Rico Qatar Réunion Romania Russian Federation Rwanda Saint Helena, Ascension and Tristan da Cunha Saint Kitts and Nevis Saint Lucia Saint Pierre and Miquelon Saint Vincent and The Grenadines Samoa San Marino Sao Tome and Principe Saudi Arabia Senegal Serbia Seychelles Sierra Leone Singapore Slovakia Slovenia Solomon Islands Somalia South Africa South Georgia and The South Sandwich Islands Spain Sri Lanka Sudan Suriname Svalbard and Jan Mayen Swaziland Sweden Switzerland Syrian Arab Republic Taiwan Tajikistan Tanzania Thailand Timor-Leste Togo Tokelau Tonga Trinidad and Tobago Tunisia Turkey Turkmenistan Turks and Caicos Islands Tuvalu Uganda Ukraine United Arab Emirates US Minor Outlying Islands Uruguay Uzbekistan Vanuatu Venezuela Vietnam British Virgin Islands US Virgin Islands Wallis and Futuna Western Sahara Yemen Zambia Zimbabwe Kosovo By downloading this case study, you acknowledge that GlobalData may share your information with Pharma Buyers Guide and that your personal data will be used as described in their Privacy Policy Submit Visit our Privacy Policy for more information about our services, how GlobalData may use, process and share your personal data, including information on your rights in respect of your personal data and how you can unsubscribe from future marketing communications. Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address. Thank you. Please check your email to download the Buyers Guide.

上市批准引进/卖出生物类似药

2023-04-18

·BioSpace

ORPHELIA Pharma Selects Tanner Pharma Group to Initiate Named Patient Program for KIMOZO® (temozolomide) in European Countries

PARIS & CHARLOTTE, N.C.--(BUSINESS WIRE)-- ORPHELIA Pharma, a French biopharmaceutical company dedicated to the development and marketing of pediatric and orphan medicines, and Tanner Pharma Group, a global provider of integrated specialty access solutions, have signed an exclusive agreement to manage the supply and distribution of Ped-TMZ, also known as KIMOZO® (temozolomide 40 mg/ml, oral suspension), to meet the special demands which may come from physicians for this unapproved pediatric form of temozolomide. Temozolomide is an anti-cancer drug approved as capsules or IV formulation in Europe for certain brain tumors. It is also used in treatment protocols for other rare cancer indications that mainly affect very young children, including refractory or relapsed neuroblastoma. In young children, an oral suspension of temozolomide is age-appropriate to ensure correct medication administration. Under the terms of the agreement, Tanner Pharma will provide access to KIMOZO® via a named patient program in European countries, in particular countries where KIMOZO® has already been used as an investigational medication in the context of ORPHELIA Pharma's now fully enrolled TEMOkids clinical trial. A named patient program is a mechanism through which physicians can legally and ethically prescribe investigational or approved drugs for patients prior to their commercial availability. The access to unapproved drugs is strictly regulated and adherence to national regulations is mandatory. Tanner Pharma will manage all elements of the supply including healthcare practitioner enquiry management, national regulatory oversight, liaison with national authorities, and logistics. The agreement currently covers all European territories but France. Healthcare professionals can obtain details about the program by contacting: kimozo@tannerpharma.com. About KIMOZO® 40 mg/ml KIMOZO® 40 mg/ml is a liquid, taste-masked and ready-to-use drinkable formulation of temozolomide developed in collaboration between the pharmacists and clinicians of Gustave Roussy hospital and the development team of ORPHELIA Pharma. KIMOZO® is being developed for the treatment of refractory and relapsed neuroblastoma, a childhood cancer of dismal prognosis. KIMOZO® was granted Early Access Authorisation (Autorisation d’Accès Précoce) by the French authorities in March 2022. The formulation of KIMOZO® is covered by a patent application in Europe and in the US. For more information about ORPHELIA Pharma, visit . For more information about Tanner Pharma Group, visit . View source version on businesswire.com: Contacts ORPHELIA Pharma Magali Rosbif Tel: +33 (0)1 42 77 08 18 orphelia@orphelia-pharma.eu Source: Tanner Pharma Group View this news release online at:

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