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Regulatory Focus™ > News Articles > 5 > Two Years of PRIME Experience: EMA Grants Eligibility to 36 Medicines

Two Years of PRIME Experience: EMA Grants Eligibility to 36 Medicines

Posted 07 May 2018 | By Zachary Brennan 

Two Years of PRIME Experience: EMA Grants Eligibility to 36 Medicines

With two years of experience under its belt with the PRIority MEdicines (PRIME) scheme, the European Medicines Agency (EMA) offered its perspective in a new report Monday on a program designed to offer additional support for biopharma sponsors developing products with promising preliminary clinical evidence and the potential to significantly address an unmet medical need.

Of the 177 requests for eligibility to PRIME received and assessed since the scheme’s launch in March 2016, EMA says it has granted eligibility to 36 programs, 30 of which are for rare diseases and 19 of which are in oncology or hematology. Advanced therapy medicines also made up 40% of products granted eligibility.

“Two years on, the Agency has already received the first three marketing authorization applications for medicines that were accepted for PRIME,” EMA said. “They are all currently are under evaluation with the first opinion expected later in 2018.”

By comparison, many of the drugs granted eligibility in PRIME have also received breakthrough or orphan designations from the US Food and Drug Administration (FDA). And for at least one medicine granted PRIME eligibility, Novartis’ Kymriah (tisagenlecleucel), FDA is one step ahead of EMA and already approved the treatment last August.


EMA said in Monday’s report that both the number of requests and the rate eligible/granted indicator have remained fairly constant and matched forecasts. With an average of eight requests received per month, EMA also said the quality of applications received is good and most eligibility requests received (161 out of 169) are at the proof of concept stage supported by exploratory data.

In terms of what additional support sponsors receive, PRIME products have received enhanced support from the agency, with so far: 31 kick-off meetings, followed by 37 scientific advice sessions (on 22 different products), with “many including input from multiple committees as well as other stakeholders (Health Technology Assessment (HTA) bodies, patients).”

Any sponsor engaged in the exploratory clinical trial phase of development can submit a request to enter the PRIME scheme, based on the availability of preliminary clinical evidence in patients indicating the promising activity of the medicinal product and its potential to significantly address an unmet medical need (proof of concept).

EMA also noted that smaller companies and academics can apply earlier to PRIME.

“While PRIME is open to all companies on the basis of preliminary clinical evidence (proof of concept), applicants from the academic sector and SMEs [small- and medium-sized enterprises] can apply earlier on the basis of compelling non-clinical data and tolerability data from initial clinical trials (proof of principle),” the agency said.

In the two years, EMA also said only eight requests were submitted by SMEs at the proof of principle stage.

“Out of these, three had been granted eligibility to PRIME at this early stage of development. Notably, the sponsor for one of these products has subsequently provided the Agency with exploratory data on its progress to proof of concept, enabling confirmation of PRIME eligibility,” the report said.
Products Granted PRIME Eligibility
Company Name/Active Substance Type Indication Supporting Data FDA Breakthrough Therapy Designation
Albireo A4250 Chemical Progressive Familial Intrahepatic Cholestasis Nonclinical + Clinical exploratory No
Spark Therapeutics/ Pfizer PF-06838435/SPK-9001 Advanced Therapy Hemophilia B Nonclinical + Clinical exploratory Yes
Biomarin BMN 270 Advanced Therapy Hemophilia A Nonclinical + Clinical exploratory Yes
uniQure AMT-060. AMT-061 Advanced Therapy Hemophilia B Nonclinical + Clinical exploratory Yes
AveXis (acquired by Novartis) AVXS-101 Advanced Therapy Pediatric patients diagnosed with spinal muscular atrophy Type 1 Nonclinical + Clinical exploratory Yes
MeiraGTx AAV2/8-hCARp.hCNGB Advanced Therapy Achromatopsia associated with defects in CNGB3 Nonclinical + Tolerability first in man No (but did receive rare pediatric disease designation)
Biogen Aducanumab Biological Alzheimer's Disease Nonclinical + Clinical exploratory No
Sage Therapeutics Allopregnanolone (Brexanolone) Chemical Postpartum depression Nonclinical + Clinical exploratory Yes
Apogenix Asunercept Biological Glioblastoma Nonclinical + Clinical exploratory No
BlueBird Bio Autologous CD34+ haematopoietic stem cells transduced with lentiviral vector encoding the human βA-T87Q-globin gene (Lentiglobin) Advanced Therapy Beta-thalassaemia major Nonclinical + Clinical exploratory Yes
Adaptimmune NY-ESO-1c259T Advanced Therapy Treatment of HLA-A*0201, HLA-A*0205, or HLA-A*0206 allele positive patients with inoperable or metastatic synovial sarcoma who have received prior chemotherapy and whose tumour expresses the NY-ESO-1 tumour antigen Nonclinical + Clinical exploratory Yes
Juno Therapeutics (acquired by Celgene) JCAR017 Advanced Therapy Relapsed/refractory diffuse large B-cell lymphoma (DLBCL) Nonclinical + Clinical exploratory Yes
Celgene and Blubird Bio bb2121 Advanced Therapy Relapsed and refractory multiple myeloma patients whose prior therapy included a proteasome inhibitor, an immunomodulatory agent and an anti-CD38 antibody Nonclinical + Clinical exploratory Yes
Novimmune Emapalumab (NI-0501) Biological Primary haemophagocytic lymphohistiocytosis (HLH) Nonclinical + Clinical exploratory Yes
Ignyta Entrectinib Chemical NTRK fusion-positive, locally advanced or metastatic solid tumours in adult and paediatric patients who have either progressed following prior therapies or who have no acceptable standard therapy Nonclinical + Clinical exploratory Yes
Alnylam Givosiran Chemical Prevention of acute attacks of hepatic porphyria Nonclinical + Clinical exploratory Yes
Edimer Pharmaceuticals EMI 200 Biological Treatment of X-linked hypohidrotic ectodermal dysplasia Nonclinical + Clinical exploratory No but obtained Fast Track and orphan designations
GlaxoSmithKline Humanized antibody targeting B cell maturation antigen conjugated with maleimidocaproyl monomethyl auristatin F (GSK2857916)  Biological Multiple myeloma Nonclinical + Clinical exploratory Yes
Hansa Medical Imlifidase  Biological Prevention of graft rejection following solid organ transplantation Nonclinical + Clinical exploratory No but obtained orphan drug designation
Inotrem LR12 (Motrem) Chemical Septic shock Nonclinical + tolerability first in man No
Alnynam Lumasiran Chemical Primary Hyperoxaluria Type 1 Nonclinical + Clinical exploratory Yes
Sanofi olipudase alfa Biological Non-neurological manifestations of acid sphingomyelinase deficiency  Nonclinical + Clinical exploratory Yes
Roche Polatuzumab vedotin Biological Relapsed and refractory patients with diffuse large B cell lymphoma Clinical exploratory Yes
Allergan Rapastinel Chemical Major depressive disorder Nonclinical + Clinical exploratory Yes
Mereo BioPharma BPS804 (Setrusumab) Biological Osteogenesis imperfecta types I, III and IV Nonclinical + Clinical exploratory No
ChemoCentryx CCX168 (Avacopan) Chemical ANCA Associated Vasculitis Nonclinical + Clinical exploratory No but granted orphan designation
Kite Pharma KTE-C19 Advanced Therapy Diffuse Large B-Cell Lymphoma Nonclinical + Clinical Exploratory Yes
Tocagen Vocimagene amiretrorepvec Advanced Therapy High grade glioma Nonclinical + Clinical Exploratory Yes
Merck Ebola Zaire Vaccine Vaccine Ebola Virus Nonnclinical + Clinical Exploratory Yes
CymaBay MBX-8025 Chemical Primary Biliary Choloangitis Nonclinical + Clinical Exploratory No
MYR GmbH Myrcludex B Chemical Treatment of chronic hepatitis D Nonclinical + Clinical Exploratory No
AtaraBio ATA 129 Advanced Therapy Epstein-Barr Virus-associated Post Transplant Lymphoproliferative Disorder in the allogeneic hematopoietic cell transplant setting who have failed on rituximab. Nonclinical + Clinical Exploratory Yes
DNAtrix Therapeutics DNX-2401 Advanced Therapy Treatment of recurrent glioblastoma in patients for which a gross total resection is not possible or advisable, or for those who refuse further surgery Nonclinical + Clinical Exploratory No but obtained Fast Track status
GBT GBT440 (Voxelotor) Chemical Sickle Cell Disease Nonclinical + Clinical Exploratory No but it obtained Fast Track


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