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The European Medicines Agency is continuing to receive a fair number of applications for a place on its PRIME (priority medicines) scheme for getting drugs for unmet needs to patients faster, but it is still rejecting most of them.

Of the 14 applications that were submitted in the first two months of 2019, only two made the grade.

AstraZeneca’s investigational monoclonal antibody, MEDI8897, for preventing lower respiratory tract infection (LRTI) caused by respiratory syncytial virus (RSV), was accepted onto PRIME in January, and Freeline’s investigational AAV gene therapy for hemophilia B, FLT180a, was accepted in February.

PRIME is popular because it offers drug developers enhanced scientific and regulatory support from the EMA and the likelihood of having their product reviewed under the EU’s accelerated assessment procedure. But since its launch in March 2016, applicants have struggled to meet the scheme’s eligibility criteria, which requires a product to target an unmet medical need and show potential benefit for patients based on early clinical data.

Of the 221 applications that the EMA has received and reviewed to date, only 50 have been accepted, and 171 have been rejected. A further eight applications have also been submitted but were not reviewed because they fell outside the scope of the scheme (e.g., the product was too early in its development or not within the EMA’s remit) and not because of anything to do with the quality of the submission.

The agency last May updated its guidance and applicant’s template to further clarify its expectations with respect to the robustness of data and applications. Eight months on, the majority of applications are still missing the mark, with the rate of products granted access to PRIME –22% –remaining stable since the launch of the scheme.

 

Why So Few Applications Are Accepted

 

“PRIME is meant for the most promising medicines and EMA focuses its attention on medicines that have the potential to bring a major therapeutic advantage,” the agency told the Pink Sheet. “That is why only a limited number of applications (22% on average) are accepted into the scheme,” it said.

The EMA is also satisfied that its guidance is fit for purpose. It said that in view of the fact that only a few applications (i.e., eight) had been found to be outside the scope of the scheme, “the guidance is considered sufficiently clear for applicants to understand the scope and eligibility criteria for PRIME.”

Applications for PRIME are understood to take time and effort to prepare, but applicants, it appears, have not been put off by the scheme’s high rejection rate. According to the EMA’s two-year report on the scheme, the number of requests has been fairly constant and has matched forecasts, with an average of eight applications received per month.

 

Essential For Developers

 

The European drug industry federation EFPIA told thePink Sheet that PRIME and a similar scheme in the US – breakthrough therapy designation (BTD), under which the Food and Drug Administration provides strengthened regulatory support during the development of drugs for serious conditions – were “essential” for developers and industry.

EFPIA noted that through PRIME, medicine developers are offered early and proactive support to optimize the generation of robust data on a medicine's benefits and risks, and they can expect to be eligible for an accelerated assessment of their medicine’s eventual marketing authorization application. “This will help patients to benefit as early as possible from therapies that target an unmet medical need and may significantly improve their quality of life.”

“We also note that the core recommendations of [the] EMA in the Regulatory Science Strategy to 2025 are to continue to promote and invest in the PRIME scheme,” EFPIA said.

In addition, EFPIA said it welcomed plans by the EMA to consider introducing a faster scientific advice process (ie within a 30-day timeframe) in PRIME, as discussed in November 2018 during the agency’s “Stakeholder workshop on support to quality development in early access approaches, such as PRIME and Breakthrough Therapies.”

The trade group has some ideas on how the scheme could be improved. It said that based on experience with PRIME at the two-year mark, it has suggested introducing the following:

 

  • an earlier entry point to the scheme based on non-clinical data – this would help to optimize the development though an iterative discussion;
  • pre-submission dialogue on eligibility and clarification on the time to entry;
  • interactive dialogue beyond the structure of the classical scientific advice process – this would benefit evidence generation via more nimble interactions with the agency’s rapporteur team and cross-committees to ensure a holistic view of the medicine’s development by all stakeholders;
  • an efficient and integrative management process to facilitate global development and patient access; and
  • a rolling submission/review system as used in other regions – this would allow better time and resource management.

AstraZeneca’s MEDI8897

 

Regarding the products that made it onto the scheme this year, MEDI8897 is an extended half-life RSV F monoclonal antibody that is being developed by AstraZeneca’s global biologics research and development arm, MedImmune.

MEDI8897 is AstraZeneca’s first product to win PRIME designation. Its acceptance on the scheme was based on the primary analysis of a Phase IIb trial which, the company said, “met its primary endpoint defined as a statistically-significant reduction in the incidence of medically-attended LRTI caused by reverse transcriptase polymerase chain reaction-confirmed RSV for 150 days after dosing in healthy preterm infants.”

According to the company, MEDI8897 is being developed for use in a broader infant population than the current standard of care for RSV prevention, AstraZeneca’s Synagis (palivizumab), “which in the EU is only approved for use in high-risk infants. Additionally, MEDI8897 is being developed so that it may only require one dose during a typical five-month RSV season vs. monthly injections with current standard of care.”

MEDI8897 has also received breakthrough therapy designation (BTD) from the FDA. (Also see "Keeping Track: CDER Approves Its First Two Novel Agents Of 2019" - Pink Sheet, 10 Feb, 2019.)

 

Freeline’s FLT180a

 

As for Freeline’s FLT180a, a corporate presentation in January stated that the advanced therapy is in a Phase I/II clinical trial, B-AMAZE, which is assessing the safety and efficacy of systemic administration of FLT180a in adults with severe hemophilia B.

FLT180a has received EU orphan drug designation. According to the EMA, the product is made of a virus that contains copies of the gene responsible for producing factor IX (FIX). “When injected into the patient, it is expected that the virus will be carried into the liver cells and start producing factor IX,” the agency said. “It is expected that a single dose of the medicine will maintain raised levels of factor IX for a long time thereby reducing bleeding.”

Freeline believes that FLT180a has the potential to be the first hemophilia B gene therapy to provide normalization of FIX levels. The company is a privately-held, clinical-stage biotechnology firm. It is based in the UK and has operations in Germany and the US.

 

The 12 Rejected Applications

 

The 12 PRIME applications that were rejected in January and February (six in each month) concerned 11 chemical drugs and one biologic. These products are being developed for a range of indications: mild cognitive impairment due to Alzheimer’s disease (AD) and mild AD dementia; Parkinson’s disease (relating to Levodopa-induced dyskinesia, Parkinson’s disease psychosis and Parkinson’s disease dementia); cystic fibrosis, cancer cachexia, hepatic injury in high-risk patients following a potentially hepatotoxic quantity of acetaminophen; advanced and/or recurrent endometrial cancer; mantle cell lymphoma; myelodysplastic syndrome; and acute exacerbations of chronic obstructive pulmonary disease. 

New applications for PRIME are reviewed during the monthly meetings of the EMA’s Committee for Medicinal Products for Human Use (CHMP). The next CHMP meeting is due to take place on March 25-28.

From the editors of Scrip Regulatory Affairs.

 

 

 

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