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

FundingEU

11.07.2014 Among the seven newly launched Joint Technology Initiatives (JTI) of Horizon2020 are two with deep roots in biotech: IMI is going into its second round while the newly established Bio-based Industries partnership invests in bioeconomy.

Stock marketNetherlandsUKBelgium

10.07.2014 Not one but two biopharma companies are seeking entry into the stock market: After Abzena raised €25m in an IPO earlier this week, Argen-x has followed suit and sold shares worth €40m.

M&AUKIreland

08.07.2014 Abbvie is determined to bring home British rare disease specialist Shire in a merger worth £30.1bn (€37.9bn). So far, Shire has rejected all offers from the US company.

M&ASwitzerland

04.07.2014 Genentech, a member of Swiss Roche Group, is set to acquire US-biotech Seragon Pharmaceuticals Inc. With the move, Genentech will obtain the rights to investigational breast cancer treatments.

M&AFranceGermany

03.07.2014 Nicox S.A. is stretching beyond the European market: The French eye health specialist is set to acquire Aciex Therapeutics Inc., a US-based pharma company with several ophthalmic therapeutics nearing market maturity.

FinanceBelgium

01.07.2014 Belgian antibody specialist Ablynx has raised €41.7m through a private placement of new shares and plans on boosting their range of nanobodies in development.

BioeconomyBelgiumUK

30.06.2014 Building on a £1.2m financing round, Celtic Renewables partners with a Belgian pilot plant to bring its bio-fuel made from whisky production residue to an industrial scale.

ConventionEUUK

25.06.2014 Sun, celebrities and a scandal - the world’s largest biotech convention is getting off to an explosive start in San Diego, California.

InvestmentGermanyNetherlands

24.06.2014 Bayer Healthcare is getting back into gene therapy with a US$252m deal aimed at the development of a haemophilia A treatment.

InvestmentNetherlands

20.06.2014 Tomtom founder Pieter Geelen invests in Dutch biotech Ocello, the company behind a 3D mapping technology used for predicting drug behaviour in patients.

Events

All Events

Current issue

All issues

Product of the week

Products

Stock list

All quotes

TOP

  • FLAMEL TECHNOLOGIES (F)14.70 USD11.28%
  • SERODUS (N)3.05 NOK7.02%
  • ADDEX (CH)4.54 CHF5.83%

FLOP

  • ANTISOMA (UK)1.32 GBP-14.84%
  • XENETIC BIOSCIENCES (UK)6.00 GBP-14.29%
  • NOVACYT (F)4.48 EUR-10.04%

TOP

  • SANTHERA (CH)70.65 CHF111.2%
  • ADDEX (CH)4.54 CHF108.3%
  • CELLECTIS (F)13.43 EUR98.1%

FLOP

  • THROMBOGENICS (B)8.67 EUR-51.8%
  • MERCK KGAA (D)63.67 EUR-50.5%
  • VERONA PHARMA (UK)1.20 GBP-43.4%

TOP

  • SANTHERA (CH)70.65 CHF3111.4%
  • GW PHARMACEUTICALS (UK)443.50 GBP843.6%
  • IXICO (UK)55.50 GBP314.2%

FLOP

  • CYTOS (CH)0.24 CHF-93.1%
  • EVOCUTIS (UK)0.21 GBP-92.6%
  • THROMBOGENICS (B)8.67 EUR-73.0%

No liability assumed, Date: 10.07.2014