First batch of drugs Primed in Europe
The European Prime programme, aimed at speeding up approval times for drugs addressing unmet needs, will soon be put to the test. The first four drugs to receive Prime status have been revealed, and encouragingly, three of them are being developed by small-to-medium enterprises (see table below).
While two of the products – Kite’s KTE-C19 and Biogen’s aducanumab – are expected to be big sellers, the other two target rare diseases and do not yet have EvaluatePharma consensus forecasts. But drugs falling into the latter group are surely the ideal candidates for Prime.
|Product||Company||Indication(s)||Status||2022e sales ($m)|
|KTE-C19||Kite Pharma||Diffuse large B cell lymphoma||Phase II||1,241|
|CCX168||ChemoCentryx||ANCA-associated vasculitis||Phase II||-|
|NI-0501||NovImmune||Haemophagocytic lymphohistiocytosis||Phase II||-|
It is not surprising to see a CAR-T candidate on the list: the products are the definition of innovative therapies that could offer an improvement over current treatments. And Kite’s KTE-C19 is arguably the most advanced in the class, with a US filing in non Hodgkin’s lymphoma (NHL) expected by the end of the year (Juno and Kite fight it out to be first to market, March 1, 2016).
The EMA has granted KTE-C19 Prime status in a type of NHL, diffuse large B-cell lymphoma (DLBCL). The product already has breakthrough therapy designation – the US equivalent of Prime – for DLBCL, primary mediastinal B cell lymphoma and transformed follicular lymphoma. It is being evaluated in all three cancers in the Zuma-1 study, with interim data due in the second half of 2016.
It is also not unexpected that cancer was one of the most popular areas for Prime applications, along with infectious diseases and pneumology/allergy.
Not every application resulted in Prime status, though – far from it. Of the 18 hopefuls since the scheme was launched in March, the EMA rejected 14.
It did not name names, but published a list of the therapeutic areas and indications they are involved in – and this suggests that KTE-C19 was not so lucky in the other NHL subtypes.
Since April 6, the agency has received a further 14 applicants for Prime, and it plans to give a monthly update on the programme.
Prime status is not a guarantee of approval – rather it is an indication that if a product can prove itself in clinical trials it could represent a significant advance.
In the case of Alzheimer’s candidate aducanumab, that is still a big if. The anti-beta-amyloid MAb is being evaluated in two identical phase III trials, Engage and Emerge, but phase I data were not exactly emphatic (Glimmers of support, but no Alzheimer’s breakthrough, July 23, 2015).
ChemoCentryx’s CCX168 is also on shaky ground. Phase III development is scheduled to start this year, but data from the phase II Clear study, while on the face of it positive, raised doubts about the drug and sent the group’s stock down 41% in January.
NovImmune’s NI-0501 looks more promising: the private Swiss company has raised $60m this year on the back of data presented at ASH (ASH – Second chances for Bluebird and Novimmune, November 17, 2015).
It might also help that the product addresses a life-threatening rare disease that mainly occurs in children, with no options other than steroids, chemotherapy and intravenous immunoglobulin.
Prime time in Europe has begun. The true test will be whether the candidates eventually make it to the market.