Another Ebola Vaccine Among New Filings At EMA

The European Medicines Agency has received 11 new marketing authorization applications, including three for orphan products from Vertex, MYR Pharmaceuticals and AstraZeneca.

Source: Shuttestock

Two New Orphan Drugs Are Being Reviewed More Quickly

The European Medicines Agency has received 11 new marketing authorization applications (MAAs) from companies seeking pan-EU approval for their products, including two applications covering Janssen’s investigational two-dose Ebola vaccine regimen, both of which have been granted fast-track review.

The other MAAs are for a range of products that include the following three orphan drugs:

• • Vertex Pharmaceuticals' triple combination regimen for cystic fibrosis, elexacaftor/tezacaftor/ivacaftor that is already approved in the US as Trikafta.
  • MYR Pharmaceuticals’ treatment for chronic hepatitis, bulevirtide (Myrcludex).
  • AstraZeneca’s acalabrutinib (Calquence), an antineoplastic medicine that was granted orphan status in the EU for treating chronic lymphocytic leukemia/small lymphocytic lymphoma.

Two of the orphan products – the triple combination regimen for cystic fibrosis and bulevirtide – were also granted accelerated assessment by the EMA. Accelerated assessments are reserved for drugs of major public health interest, particularly from the point of view of therapeutic innovation. They can cut the time it takes the EMA to evaluate an MAA from up to 210 days to up to 150 days (not counting clock stops when applicants have to provide additional information).

Among the products covered by the other new MAAs are:

• • Fluticasone propionate/salmeterol xinafoate for obstructive airway diseases.
  • Sanofi’s quadrivalent influenza vaccine Flublok (rDNA).
  • Sanofi’s meningococcal group A, C, W135 and Y conjugate vaccine.

The remaining MAAs are for two generic medicines – caffeine citrate, a psychoanaleptic, and the cancer drug sunitinib – and one biosimilar, the antidiabetic insulin aspart submitted by Biocon/Mylan.

With the exception of Janssen’s Ebola vaccine, all the applications are included in the EMA’s latest list of MAAs under review, which was compiled on 4 November. Janssen announced on 7 November that it had submitted its applications to the agency.

New Ebola Vaccine Regimen

Janssen has submitted two MAAs in parallel for a two-dose regimen – Ad26.ZEBOV and MVA-BN-Filo –  for the prevention of Ebola Virus Disease caused by Zaire ebolavirus species.

The regimen involves administering Ad26.ZEBOV as the first dose and MVA-BN-Filo as the second dose, approximately eight weeks later.

It has been used on an experimental basis in the Democratic Republic of Congo, alongside Merck Sharp & Dohme’s V920 (rVSV-ZEBOV) vaccine, which was recommended for conditional approval by the EMA’s drug evaluation committee, the CHMP, last month. (Also see "Janssen’s Ebola Vaccine Added To Fight Against African Outbreak" - Pink Sheet, 25 Sep, 2019.) (Also see "First Ebola Vaccine Among Seven Products To Get EMA Nod" - Pink Sheet, 18 Oct, 2019.)

On 31 October, Janssen announced that it would provide up to 500,000 regimens of the vaccine for use in a new clinical trial being organized by the DRC government and global health stakeholders in an effort to contain the Ebola outbreak in the country.

In the US, the company is in discussions with the Food and Drug Administration about defining the data set required to file a marketing application for the vaccine regimen under the Animal Rule licensure pathway. Janssen said it was also working with the World Health Organization to enable the registration of the vaccine regimen in African countries.

Triple Combination CF Treatment

Vertex’s triple combination treatment for CF was approved in the US as Trikafta in October for patients aged 12 years and older. The drug had been included in all available FDA programs designed to speed up approval, including priority review, fast track and breakthrough therapy designation. (Also see "Keeping Track: Vertex’ Trikafta Speeds To US Approval; New Indications For AZ’s Farxiga, J&J’s Stelara, GSK’s Zejula" - Pink Sheet, 24 Oct, 2019.)

The EU application for the treatment is supported by previously disclosed positive results from two global Phase III studies in people with cystic fibrosis, the company said. Both these studies showed statistically significant improvements in lung function, which was the primary endpoint, and in all key secondary endpoints.

Vertex told the Pink Sheet that it was “delighted to have received accelerated assessment” for its elexacaftor, tezacaftor and ivacaftor triple combination regimen by the EMA, and that it would work closely with the agency to achieve approval. As for what the triple combination would be called in the EU, the company said it was too early to comment and that the name would be announced in due course.

The Pink Sheet’s two trackers – New Filings At The EMA and EU Accelerated Assessment – will be updated in due course to reflect these latest developments.