Safe and potent isn’t enough
Enlarge image


Safe and potent isn’t enough

05.11.2012 - The European Commission declined a market authorisation for the orphan drug Elelyso in spite of a positive risk-benefit assessment.

The drug couldn’t be approved due to specific requirements of the European Union (EU) Orphan Drug Regulation. Pfizer Inc. and Israel’s Protalix BioTherapeutics, Inc. developed taliglucerase alfa, as an enzyme replacement therapy (ERT) for the treatment of Gaucher disease. They filed in November 2010 for marketing approval in the EU. In June 2012 the CHMP issued its opinion on the plant cell-expressed recombinant form of human glucocerebrosidase, stating that it could not recommend the drug for approval, because Irish drugmaker Shire’s velaglucerase alfa had received prior Marketing Authorisation with orphan drug designation for the same condition. Therefore, Shire's treatment has orphan market exclusivity in the EU for a ten-year period commencing on its authorisation in August 2010. Pfizer pursued a request for derogation from Shire's orphan market exclusivity based on a number of factors. In detail, Pfizer had been seeking an exemption to orphan exclusivity on the basis of an "insufficient supply" of enzyme replacement therapy for Gaucher's disease and the drug’s expected "major contribution to patient care." But the reasoning of the pharma giant was not enough to convince the CHMP. In USA, Australia, and Japan, Genzyme's orphan drug Cerezyme dominates the Morbus Gaucher market.

"We are disappointed by the EC's decision on taliglucerase alfa and believe it is important, given the history of past shortages, for the Gaucher disease community in the EU to have a third treatment option available," said Diem Nguyen, General Manager, Pfizer Biosimilars. "We will continue to work closely with our partner, Protalix, to make taliglucerase alfa available to the Gaucher disease community in other countries." Taliglucerase alfa was approved by the FDA in May 2012 for the long-term ERT of adults with a confirmed diagnosis of Type 1 Gaucher disease and was approved by Israel's Ministry of Health in September 2012.



09.10.2015 The US pricing controversy is reverberating halfway across the globe. Shield Therapeutics has taken its IPO plans off the table. Market conditions are too volatile, the company says.


07.10.2015 T-cell specialist Adaptimmune Therapeutics plc needs more space for its R&D in immuno-oncology. The expanding UK company is building a major facility that will house 200 staff.

Drug DiscoveryGermany

06.10.2015 Evotec has been hired by the US National Cancer Institute (NCI) to provide compound management services to the Chemical Biology Consortium. The goal: the discovery of new cancer treatments.

Drug DevelopmentUKBelgium

01.10.2015 Autoimmune disease specialist Apitope has raised €12m in a Series B financing round and is relocating its UK headquarters to Wales.

Drug DevelopmentFranceEUFinlandSpainItalyAustria

01.10.2015 Paris-based BEAM Alliance, a network of companies working on novel antibiotics founded earlier this year, has released its first position paper.


29.09.2015 Swiss CNS specialist Asceneuron SA has raised CHF30m (€27.5m) in a Series A financing, winning Paris-based Sofinnova Partners as its largest investor.


28.09.2015 US company Aduro Biotech has acquired Dutch monoclonal antibody discovery and development firm BioNovion for €29m plus milestones.


24.09.2015 Nutrition company Nestlé is teaming up with biopharmaceutical company AC Immune SA to develop a Tau diagnostic assay for early Alzheimer’s.


22.09.2015 Bayer has taken another step in its restructuring and development as a life sciences company after splitting from all its chemical divisions.


18.09.2015 AstraZeneca has partnered up with the US Department of Health in order to accelerate the development of new antibiotics. The goal: to be prepared for multi-resistant bugs – and for bioterrorism.


All Events

Current issue

All issues

Product of the week


Stock list

All quotes


  • PHARMING (NL)0.31 EUR19.23%


  • THERAMETRICS (CH)0.04 CHF-33.33%
  • BAVARIAN NORDIC (D)35.60 EUR-8.48%


  • THROMBOGENICS (B)4.13 EUR45.4%
  • OREXO (S)66.00 SEK42.5%
  • ZEALAND PHARMA (DK)156.00 DKK26.8%


  • THERAMETRICS (CH)0.04 CHF-33.3%
  • GALAPAGOS (B)38.43 EUR-27.0%


  • KARO BIO (S)43.60 SEK5282.7%
  • CHRONTECH PHARMA (S)0.35 SEK337.5%
  • GALAPAGOS (B)38.43 EUR233.9%


  • NEOVACS (F)0.70 EUR-77.3%
  • BIOTEST (D)19.15 EUR-74.6%
  • EVOCUTIS (UK)0.16 GBP-70.9%

No liability assumed, Date: 09.10.2015