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.

Perspective

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-A0201, HLA-A0205, 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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