News

EMA rejects Santhera’s Raxone
Enlarge image

RegulatoryEUSwitzerland

EMA rejects Santhera’s Raxone

21.01.2013 - The EMA has withdrawn an MAA of Switzerland’s Santhera for Raxone, a synthetic coenzyme Q10 derivative against hereditary blindness.

The EU regulatory authority argued that Santhera had investigated too few patients with Leber's Hereditary Optic Neuropathy during clinical development. However, as in the case of uniQure’s currently approved orphan drug Glybera, Santhera plans to ask the EMA’s Committee for Medicinal Products for Human Use (CHMP) to reconsider its treatment based on the clinical need the drug addresses. Only a narrow majority of CHMP had voiced concerns about the reliability of the results.

 "Since there were no concerns expressed about the safety of Raxone and the observed treatment benefit on visual acuity in these patients was clinically relevant and consistent with the published literature, we believe that we should be allowed to address the remaining concerns through further confirmatory clinical work to be conducted post-approval," Santhera CEO Thomas Meier said in a statement.

LHON is caused by mutations in mitochondrial DNA. The genetic disorder typically presents in young men, as loss of vision in both eyes, leading to blindness within a few months after the onset of symptoms. More than 95% of patients harbour one of three pathogenic mutations of the mitochondrial DNA, which cause a defect in complex I of the mitochondrial respiratory chain. The defect leads to decreased ATP production, increased oxidative stress and retinal ganglion dysfunction which cause progressive loss of visual acuity and blindness. Raxone (idebenone), a synthetic short-chain benzoquinone and a cofactor for the enzyme NAD(P)H:quinone oxidoreductase (NQO1) is capable of transferring electrons directly onto complex III of the mitochondrial electron transport chain, thereby circumventing the complex I defect.

© eurobiotechnews.eu/tg

http://www.european-biotechnology-news.com/news/news/2013-01/ema-rejects-santheras-lhon-drug.html

BusinessBelgiumSpain

31.07.2015 Spanish business developer Genetrix has sold its clinical stage cell therapy development subsidiary Coretherapix to Belgian TiGenix in a deal worth up to €267m.

BusinessUKSwitzerland

29.07.2015 Newly founded Mereo BioPharma has acquired three mid-stage clinical assets from Novartis. In turn, the Swiss pharma concern will hold a stake in the UK company. Other investors are also on board.

M&AIreland

28.07.2015 Irish Allergan is bolstering its product pipeline with a takeover of CNS specialist Naurex. At the same time, the company is divesting its global generics business to Teva.

Public marketGermanyFranceBelgiumFinlandDenmarkUK

24.07.2015 Biotech companies across Europe continue to raise cash on the public markets, generating €3bn in total financing proceedings in the first half of 2015. Compared to 2014, this is almost twice as much.

FinanceFrance

23.07.2015 US money keeps flowing into Europe's biotechs. As the race for the peanut allergy market heats up, NASDAQ listed French biotech DBV has announced the successful closing of US$281m (€256m) share offering for its allergy products.

BioplasticsFrance

21.07.2015 French biotech Deinove has launched a R&D programme to produce the versatile chemical intermediate muconic acid by using a designer variant of the Deinococcus bacteria.

BusinessFrance

16.07.2015 Sanofi has announced plans to restructure its business in five standalone global business units starting in January 2016. The goal is to promote growth and help the company launch a catalogue of new drugs over the next five years.

R&DUK

15.07.2015 GlaxoSmithKline (GSK) has become the first drugmaker to join the Francis Crick Institute. It is a consortium made up of six scientific and academic institutes to conduct research and drug development in the UK. The new institute is set to become a world-leading centre of biomedical research and innovation.

FundingUK

13.07.2015 UK drug developer ReNeuron has pulled in £68.4m (€96m) for its cell therapy pipeline for stroke and blindness treatments. Biotech guru Neil Woodford increases his fund’s stake in ReNeuron.

BusinessSweden

10.07.2015 Karolinska Development has slashed the reported value of its portfolio by almost half by revising its valuation model for early stage companies and divesting its shares in pain med maker Pharmanest.

Events

All Events

Current issue

All issues

Product of the week

Products

Stock list

All quotes

TOP

  • SERODUS (N)2.60 NOK4.00%
  • SUMMIT (UK)4.03 GBP3.87%
  • FLAMEL TECHNOLOGIES (F)23.88 USD3.83%

FLOP

  • BASILEA (CH)103.50 CHF-13.10%
  • ZELTIA (E)3.69 EUR-8.44%
  • DIAMYD MEDICAL -B- (S)9.20 SEK-6.60%

TOP

  • IXICO (UK)34.00 GBP44.7%
  • DBV TECHNOLOGIES (F)78.26 EUR40.4%
  • ZEALAND PHARMA (DK)149.00 DKK31.9%

FLOP

  • BIOTEST (D)25.40 EUR-64.2%
  • BIOTECH PHARMACON (N)9.74 NOK-43.0%
  • TRANSGENE (F)2.81 EUR-25.1%

TOP

  • ADOCIA (F)88.47 EUR590.6%
  • FORMYCON (D)28.89 EUR330.6%
  • DBV TECHNOLOGIES (F)78.26 EUR312.3%

FLOP

  • CHRONTECH PHARMA (S)0.01 SEK-88.9%
  • NEUROVIVE PHARMACEUTICAL AB (S)22.00 SEK-70.8%
  • NEOVACS (F)1.02 EUR-68.6%

No liability assumed, Date: 31.07.2015