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

FinancingSwedenNorway

31.10.2014 Another biotech is leaving the European market place in favour of a US IPO. Swedish Cortendo is now planning to go public in the US after raising €8.8m in bridge financing.

FinancingUK

29.10.2014 British VC fundraiser Advent Life Sciences has raised €184m with its second fund. The money will help seed and build life sciences companies in the UK, Europe and the US, the company said.

BusinessSwitzerlandGermanyUK

28.10.2014 Swiss pharma company Novartis is selling its influenza vaccines business to Australian biopharma specialist CSL Ltd for US$275m.

PoliticsSwitzerland

23.10.2014 Just one day before its planned initial listing, Swiss Molecular Partners has cancelled its IPO. The reason: "unfavourable market conditions".

PoliticsEU

23.10.2014 Jean-Claude Juncker can breathe a sigh of relief as MEPs voted “yes” by a large majority to his team of European Union Commissioners. Previous to the vote, Juncker had shifted the EMA back to the health commissioner in response to protests.

FinancingFrance

21.10.2014 Yet another European company is looking for money on the US stock exchange – French DBV Technologies SA is all set up for its secondary listing on NASDAQ. The French biotech company hopes to rake in US$98m for its peanut allergy patches.

FinancingDenmark

18.10.2014 After raising US$220.5m (€172m) with its IPO, fast Forward Pharma’s lucky streak hits an obstacle on the road to success with shares falling 17% on its first day of trading.

M&AUKItaly

17.10.2014 Abandoned at the altar: Abbvie has dropped plans to acquire UK’s Shire following new US tax rules. This puts a stop to one of the biggest pharma mergers planned this year.

FinancingUKBelgium

15.10.2014 Bicycle Therapeutics wins €25m backing by major pharma venture capitalists for the development of a new class of oncology drugs.

FinancingGermanySwitzerlandNetherlands

13.10.2014 Biotechnology IPOs are back in fashion – German Probiodrug AG wants to raise €32m with its IPO, whilst Swiss Molecular Partners AG hopes the launch of its IPO will gain a revenue of €145m.

Events

All Events

Current issue

All issues

Product of the week

Products

Stock list

All quotes

TOP

  • THERAMETRICS0.08 CHF14.29%
  • CYTOS0.17 CHF13.33%
  • MORPHOSYS75.99 EUR4.89%

FLOP

  • EPIGENOMICS4.15 EUR-4.38%
  • EVONIK26.20 EUR-2.42%
  • PAION2.80 EUR-2.10%

TOP

  • BIOFRONTERA2.82 EUR28.2%
  • HBM91.50 CHF8.6%
  • EPIGENOMICS4.15 EUR8.4%

FLOP

  • MAGFORCE5.55 EUR-19.6%
  • CO.DON2.65 EUR-15.6%
  • PAION2.80 EUR-13.0%

TOP

  • SANTHERA89.00 CHF2318.5%
  • CO.DON2.65 EUR167.7%
  • WILEX2.25 EUR81.5%

FLOP

  • CYTOS0.17 CHF-95.7%
  • MERCK KGAA71.68 EUR-41.5%
  • 4SC0.98 EUR-41.0%

No liability assumed, Date: 30.10.2014