News

Treating the cause
Enlarge image

ResearchUKEU

Treating the cause

29.05.2012 - A positive recommendation by EMA's CHMP committee raises hope for patients with cystic fibrosis in Europe.

London – The EU agency recommended Kalydeco (ivacaftor) from the US-biotech Vertex for approval. The treatment targets patients from the age of six years on who have a G551D mutation in the cystic fibrosis transmembrane regulator (CFTR) gene.

Kalydeco was already approved by the FDA at the end of January this year. According to the European agency, Kalydeco offers an innovative therapeutic approach for patients with cystic fibrosis: it is the first treatment that targets the underlying mechanism of the disease, by restoring the function of the mutated CFTR protein.

Currently available therapies for patients with cystic fibrosis only address the consequences of the disease, not the underlying defect. Clinical studies showed that Kalydeco improved pulmonary function in cystic fibrosis patients with the specific G551D-CFTR mutation. About half of the world's CF patients reside in Europe, where about 35,000 people have the disease. The European Commission will consider the CHMP's recommendation in its final decision on Kalydeco. According to PM live, the European recommendation comes almost four months after Kalydeco was cleared by the US Food and Drug Administration (FDA), with the US agency taking just three months to review Vertex' marketing application. This backs outcomes from a recent report that described how the FDA had a faster approval process than the EMA.

Update: After releasing this news, Vertex corrected trial data of Kalydeco.

http://www.european-biotechnology-news.com/news/news/2012-02/treating-the-cause.html

Swiss Biotech DaySwitzerland

16.04.2015 The Swiss biotech industry can look back on 2014 as a good year, but political uncertainties do remain. This was one of the topics discussed during the Swiss Biotech Day in Basel, which attracted more than 400 participants.

EventEUFrance

16.04.2015 The changing landscape in healthcare was just one of the hotly discussed topics of the 27th Annual EuroMeeting of the international association DIA.

R&DBelgium

14.04.2015 Belgian Thrombogenics NV has launched a new oncology company with Gent-based life sciences institute VIB. The new venture will develop an antibody for the treatment of a fast-growing brain tumour that affects children and adolescents.

Stock MarketsEUFranceUKNorwaySweden

09.04.2015 The European public markets remain highly attractive for biotech companies. Twice as many companies went public in the first quarter of 2015 than during the same period last year.

FinancingAustriaUKSwitzerlandGermany

08.04.2015 To bring its lead product of a new class of antimicrobials to phase 3 trials, Nabriva Therapeutics has completed a US$120m Series B financing.

Research & DevelopmentNetherlands

07.04.2015 Gene therapy is attracting more and more big pharma companies. Now, Bristol-Myers Squibb has forged a US$1bn collaboration with Dutch uniQure, also picking up a 4.9% share in the company.

Stock MarketsFrance

02.04.2015 Sensorion is the latest French biotech to go public, following a spate of IPOs on Paris Euronext. The biopharma plans for a €12m IPO to finance its pipeline of inner ear disease drugs.

M&AIreland

31.03.2015 In its aim to expand its drug portfolio in orphan disease products, Horizon Pharma has acquired US company Hyperion Therapeutics for US$1.1bn in cash. The takeover entitles the UK biopharma to all of Hyperion’s common stock.

BusinessGermany

31.03.2015 Merck Serono has forged a US$941m (€876m) deal with US Intrexon for the development and commercialisation of CAR-T therapies. The new CAR-T projects are set to strengthen their R&D technology portfolio in immuno-oncology.

Stock MarketsFranceEU

27.03.2015 Europe is once more proving that it can be a good bet for biotech companies going public. A newly established EU-wide lobbying initiative could help the IPO boom on its way.

Events

All Events

Partner-Events

München

BioVaria 2015

Current issue

All issues

Product of the week

Products

Stock list

All quotes

TOP

  • MEDIGENE14.50 EUR9.19%
  • SARTORIUS143.75 EUR7.28%
  • FORMYCON31.50 EUR4.06%

FLOP

  • THERAMETRICS0.08 CHF-11.11%
  • CO.DON2.90 EUR-7.94%
  • 4SC1.25 EUR-6.72%

TOP

  • MEDIGENE14.50 EUR117.4%
  • 4SC1.25 EUR76.1%
  • FORMYCON31.50 EUR39.9%

FLOP

  • CYTOS1.27 CHF-15.3%
  • BB BIOTECH280.70 EUR-11.5%
  • MORPHOSYS62.50 EUR-10.2%

TOP

  • SANTHERA99.75 CHF2709.9%
  • CYTOS1.27 CHF647.1%
  • FORMYCON31.50 EUR343.7%

FLOP

  • MOLOGEN5.02 EUR-53.5%
  • BIOFRONTERA2.60 EUR-20.2%
  • THERAMETRICS0.08 CHF-20.0%

No liability assumed, Date: 17.04.2015