EMA recommends two orphan drugs for approval

The CHMP recommended granting a marketing authorisation for Ammtek’s orphan drug Amglidia (glibenclamide) for the treatment of neonatal diabetes mellitus in newborns, infants and children.

Pfizer Ltd  received a recommendation for its cancer antibody drug conjugate (ADC) Mylotarg (gemtuzumab ozogamicin) in combination with the chemotherapeutics cytarabine and daunorubicin for the treatment of CD33-positive acute myeloid leukaemia (AML), an orphan blood cancer, in treatment-native patients older than 15 years. Mylotarg couples a CD33-targeting antibody moiety to the minor groove binder calicheamicin, a toxic payload originally derived from the bacterium Micromonospora echinospora in the mid-1980s. In September 2017, the US Food and Drug Administration approved Mylotarg  for the treatment of treatment-native CD33-positive adult and refractory AML patients over 2 years of  age at a lower dose than as the original monotherapy, which had got accelerated approval in May 2000 for relapsed adult patients. Mylotarg was then voluntarily withdrawn from the market after subsequent confirmatory trials failed to verify clinical benefit and demonstrated safety concerns, including a high number of early deaths.

The Committee also recommended Alpivab (peramivir), a FDA-approved neuraminidase blocker developed by BioCryst Pharmaceuticals Inc for the treatment of uncomplicated influenza.

The CHMP rejected to recommend EU market approval of Puma Biotechnology’s Nerlynx (neratinib), an oral blocker of HER1, HER2 and HER4 kinases, for adjuvant treatment of Her2-positive early stage breast cancer.

The CHMP also adopted a negative opinion for an extension of therapeutic indication for Pfizer Ltd’s approved GIST, pancreatic neuroendocrine tumour and metastatic renal cell carcinoma drug Sutent (sunitinib) because it found the evidence that Sutent delays the return of the cancer was not convincing.

Furthermore, Gilead Sciences International Ltd withdraw an application to extend the use of Zydelig (idelalisib) the treatment of chronic lymphocytic leukaemia. Whilst the study met the primary endpoint for progression-free survival and demonstrated a clinically meaningful benefit in overall survival, according to Gilead, the EMA requested longer term data „to allow the committee to conclude on a positive benefit-risk evaluation for the requested indication.“