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

AwardFinlandEU

27.05.2016 The Finnish Technology Academy has awarded US-American innovator Frances Arnold the 2016 Millennium Technology Prize, worth €1m. Arnold is a pioneer in the field of directed evolution.

OncologyUK

26.05.2016 BigDNA relaunches as Iceni Pharmaceuticals with the aim to develop repurposed and reformulated cancer therapies. First order of business: repurpose Merck Serono’s cilengitide as a multiple myeloma treatment.

MicrobiomeFranceDenmarkEU

24.05.2016 One of the pioneering companies developing pharmaceuticals and diagnostics based on the gut microbiome, Enterome Bioscience, has raised €14.5m in a Series C financing round. Among the investors were Seventure and Lundbeckfond as well as Nestlé.

AntibioticsEUUKItaly

20.05.2016 The long awaited global review on antimicrobial resistance by economist Lord Jim O’Neill has been published. It sets out an action plan to defeat superbugs with a huge awareness campaign and rapid diagnostics to be used before antibiotics are prescribed.

LicensingGermanyIrelandSwitzerland

18.05.2016 Bayer is deepening its involvement in CRISPR with a licensing agreement for genome editing patents. Irish partner ERS Genomics holds the rights to the CRISPR/Cas9 tech from Emmanuelle Charpentier, one of the inventors.

M&AGermany

17.05.2016 Newly rebranded Siemens Healthcare has entered the field of molecular testing for cancers with the acquisition of Cologne-based NEO New Oncology.

FinancingUK

11.05.2016 Newly spun out company OxStem has raised £16.9m (€21.5m) to develop regenerative meds for the treatment of age-related disease. It is the largest financing for an Oxford spin-out – or any UK academic spin-out – to date.

FinancingSwitzerland

09.05.2016 Swiss Genentech partner AC Immune has raised CHF42.7m (€38.6m) in order to advance its therapeutic and diagnostic product pipeline in Alzheimer’s disease. The news follows a recently announced R&D collaboration with Biogen.

FinancingSpain

04.05.2016 Barcelona-based epigenetics expert Oryzon Genomics has closed a debt funding round of €10.5m, bringing the total money raised since last year to €27m. The funds will serve to advance the company’s two LSD1 inhibitors in cancer and neurodegeneration.

Open InnovationFrance

03.05.2016 Ipsen is strengthening its ties to long-time development partner Oncodesign, a Dijon-based cancer treatment biotech. The French pharma is handing over the pre-clinical pharmacology for its oncology research programmes to Oncodesign.

Quick news provided by GlobeNewswire

View more stock news

Events

All Events

Partner-Events

Aachen

Biomedica 2016

Basel (CH)

Chemspec Europe 2016

Current issue

All issues

Product of the week

Products

Stock list

All quotes

TOP

  • BIOTIE THERAPEUTICS (FI)0.27 EUR92.86%
  • PLETHORA (UK)3.38 GBP12.67%
  • ALMIRALL (E)14.43 EUR7.93%

FLOP

  • 4SC (D)2.59 EUR-20.31%
  • NEUROVIVE PHARMACEUTICAL AB (S)5.25 SEK-4.55%
  • FORMYCON (D)20.15 EUR-4.37%

TOP

  • GALAPAGOS (B)51.86 EUR30.7%
  • NICOX (F)11.00 EUR28.7%
  • GENMAB (DK)1177.00 DKK22.2%

FLOP

  • MOBERG PHARMA (S)38.00 SEK-21.8%
  • 4SC (D)2.59 EUR-21.5%
  • FORMYCON (D)20.15 EUR-18.0%

TOP

  • KARO BIO (S)30.60 SEK1981.6%
  • CHRONTECH PHARMA (S)0.34 SEK1600.0%
  • NICOX (F)11.00 EUR491.4%

FLOP

  • NEUROVIVE PHARMACEUTICAL AB (S)5.25 SEK-89.0%
  • BB BIOTECH (D)44.03 EUR-84.5%
  • BIOTEST (D)17.15 EUR-78.1%

No liability assumed, Date: 29.05.2016