News

Safe and potent isn’t enough
Enlarge image

RegulatoryEU

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.

© eurobiotechnews.eu/bk

http://www.european-biotechnology-news.com/news/news/2012-04/safe-and-potent-isnt-enough.html

BusinessGermany

19.09.2014 In order to concentrate on the life science business, German industry giant Bayer is splitting off its plastics subgroup as a separate company.

FinanceSwitzerland

18.09.2014 Swiss biopharma Cardiorentis has received a €45m investment from overseas investment firm Healthcare Royalty Partners in order to finance the registration for its heart drug.

PoliticsEU

15.09.2014 Jean Claude Juncker, the European Commission President-elect revealed his new Commissioners last week, whilst announcing a change in the units dealing with medicines, medical devices and health technology.

BiosimilarsGermanyFrance

12.09.2014 The 2011 collaboration of Boehringer Ingelheim and Eli Lilly is bearing fruit. An insulin biosimilar developed through the partnership has now been approved in Europe - the first-ever generic version of this class.

FinancingNetherlands

11.09.2014 Cystic fibrosis specialist Proqr Therapeutics has set the terms for its upcoming IPO at US-American stock exchange NASDAQ.

Clinical TrialsUKSweden

09.09.2014 In the midst of the hustle and bustle of the European Respiratory Society’s International Congress 2014, Astrazeneca quietly announced the disappointing results of its latest benralizumab study.

BusinessDenmark

04.09.2014 After their arthritis product’s Phase IIb failure, Novo Nordisk A/S is pulling out of all research and development activities within inflammatory disorders.

BusinessBelgiumIrelandSwitzerland

02.09.2014 In the face of declining revenue, Belgian biopharmaceutical company Thrombogenics has decided to spin out its cancer research and development activities.

ResearchUKSwitzerlandItaly

29.08.2014 Just a week ago, Glaxosmithkline defused the WHO’s claim that an Ebola vaccine could be ready by 2015. Now, the British company’s own candidate vaccine is to be rushed to human trials with the help of funding from an international consortium.

Events

All Events

Current issue

All issues

Product of the week

Products

Stock list

All quotes

TOP

  • THERAMETRICS0.09 CHF12.50%
  • MERCK KGAA72.63 EUR4.35%
  • MAGFORCE7.30 EUR4.29%

FLOP

  • CYTOS0.21 CHF-8.70%
  • WILEX2.22 EUR-5.93%
  • CO.DON3.08 EUR-5.81%

TOP

  • PAION3.25 EUR31.0%
  • CO.DON3.08 EUR23.2%
  • EPIGENOMICS3.80 EUR22.6%

FLOP

  • CYTOS0.21 CHF-19.2%
  • ADDEX3.25 CHF-18.8%
  • MEDIGENE3.86 EUR-17.0%

TOP

  • SANTHERA89.30 CHF2088.7%
  • CO.DON3.08 EUR234.8%
  • PAION3.25 EUR221.8%

FLOP

  • CYTOS0.21 CHF-94.8%
  • BIOFRONTERA2.24 EUR-38.6%
  • 4SC1.14 EUR-38.4%

No liability assumed, Date: 22.09.2014