Round-up: CHMP backs two potential orphan blockbusters

Drug recommendations from the monthly meeting of Europe’s CHMP advisory committee were delayed until late on Friday because of the Easter break.

But it was clearly a busy meeting for Europe’s regulators, with several key drugs backed for marketing authorisation. The CHMP recommended a conditional marketing authorisation for Janssen’s Darzalex (daratumumab) in multiple myeloma.

Despite many competitors it has already been approved in the US and is tipped as a potential blockbuster, after being developed by Denmark’s Genmab.

Darzalex is to be used in patients with relapsed or refractory disease whose previous treatment included a proteasome inhibitor and an immunomodulatory agent and whose disease worsened after treatment.

As part of the conditional marketing authorisation, Janssen must provide results from two Phase 3 studies of Darzalex used in combination with standard treatments for this disease (lenalidomide/dexamethasone and bortezomib/dexamethasone).

Both studies are ongoing and the data will be provided by the second half of 2017. Until data are available, the CHMP will review Darzalex annually to determine whether the conditional marketing authorisation can be maintained.

Darzalex, an orphan drug, is the first CD38-directed monoclonal antibody recommended for approval in Europe. It triggers the patient’s own immune system to attack cancer cells and resulting in tumour cell death.

The CHMP also recommended another orphan drug tipped for billion-plus sales – Amicus therapeutics’s Galafold (migalastat) oral drug for Fabry disease.

Chardan Capital forecasts 2025 sales to be around $1.21 billion. Unlike existing enzyme replacement therapies, such as Genzyme’s Fabrazyme (agalsidase beta) and Shire’s Replagal (agalsidase alfa), Galafold acts as a “pharmacological chaperone”.

It binds to defective alpha-galactosidase A enzyme, allowing it to be transported to where its action is needed and restore its activity.

The regulators have backed a change of label for Janssen’s paliperidone palmitate schizophrenia for schizophrenia, allowing it to be administered four times a year.

Formerly known as Paliperidone Janssen, it will be marketed under the brand name, Trevicta.

GlaxoSmithKline’s Strimvelis, an advanced cell therapy was backed for severe combined immunodeficiency due to adenosine deaminase deficiency, a rare inherited disorder.

The autologous CD34+ cells join with bone marrow cells and express the pharmacologically active enzyme.

Also recommended by the CHMP was AstraZeneca unit MedImmune’s H5N1 pandemic flu vaccine. This is the first bird flu vaccine to be recommended for approval in the EU.

The CHMP also gave a positive opinion for an earlier use for AbbVie’s huge selling Humira (adalimumab), which can now be used in moderate paediatric Crohn’s disease instead of only severely active Crohn’s, after failure or intolerance to a nutrition therapy and a corticosteroid or an immunomodulator.

Also Eisai’s Halaven cancer drug was backed in unresectable liposarcoma, who have received prior anthracycline containing therapy for advanced or metastatic disease.

Bristol-Myers Squibb’s cancer immunotherapy Opdivo (nivolumab) was backed for a new indication in renal cell carcinoma, and can also be used in all metastatic non-small cell lung cancer after chemotherapy, instead of just squamous disease.

As anticipated by pharmaphorum, the CHMP also gave the green light to a second biosimilar of MSD’s inflammatory diseases drug, Remicade (infliximab) – Samsung Bioepis’s Flixabi (infliximab). It also backed a generic version of Eisai’s Aloxi (palonosetron) cancer vomiting drug, from Accord Healthcare.

All positive opinions will be passed to the European Commission, which will likely grant marketing authorisations in the coming weeks.

In drug safety, the CHMP is reviewing the antibiotic vancomycin to update the label and prevent build-up of resistance. It is reviewing Symbiopharm’s Symbioflor 2 E. coli-containing medicine after the German regulator raised concerns about its effectiveness.

Related articles:

Myeloma drug Kypolis launched in the UK

Genzyme in breach of ABPI code over Fabry drug

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