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21.01.2013 - The EMA has withdrawn an MAA of Switzerland’s Santhera for Raxone, a synthetic coenzyme Q10 derivative against hereditary blindness.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

FinancingPoland

24.03.2015 Poland has announced the closing of a new venture capital fund at US$42m (€32m). The fund, which is the first of its kind in Poland, is dedicated to the Life Sciences with a strong connection to Central and Eastern Europe.

BioeconomyFranceSwedenGermanyNetherlandsFinland

23.03.2015 A BASF-led consortium aims to decrease bio-based production costs using industrial biotechnology. The Horizon2020 funded consortium is the sixth project of the SPIRE Public Private Partnership and has a budget of €14m.

Stock MarketsFranceSwedenUKNorwayIreland

20.03.2015 There are no signs of inertia creeping into the European biotech industry this spring. A total of eight IPOs are set to go public by the end of the year’s first quarter.

FinancingUKEU

17.03.2015 Pharma giants GlaxoSmithKline (GSK) and Johnson & Johnson (J&J) will be key collaborators in a new global fund for dementia research to address the rising threat posed by dementia and Alzheimer’s disease.

FinancingFranceDenmarkSwedenGermany

19.03.2015 French venture capital firm Seventure Partners has raised a further €25m for its microbiome-focused Health for Life Capital Fund. It now stands at €100m.

EventsFranceEUSwitzerlandUKGermany

12.03.2015 With around 1,400 companies and 2,400 participants, the springtime counterpart of BIO-Europe enjoyed a successful premiere in France this year. The overall mood of the European biotech industry was positive, and the French hosts in particular reported new initiatives.

M&ABelgium

11.03.2015 Belgian cell-based therapeutics developer and CMO MaSTherCell SA has been acquired by US biotech Orgenesis Inc. for €23m.

FinancingGermany

10.03.2015 The Gates Foundation has dipped into its purse to bring infectious diseases vaccines to the poorest countries. German vaccine specialist CureVac is set to receive US$52m for their mRNA platform technology.

Stock MarketsIrelandUK

05.03.2015 The IPO wave in the biotech sector shows no signs of slowing down. An Irish life sciences fund set up by former Elan executives plans to raise €325m with its IPO, whilst Summit Therapeutics has announced its IPO pricing of US$34m on Nasdaq.

LicencingDenmark

05.03.2015 Bavarian Nordic is outlicensing its Phase 3 prostate cancer immunotherapy Prostvac to pharma heavyweight Bristol Myers Squibb for a tidy €881m.

Events

All Events

Current issue

All issues

Product of the week

Products

Stock list

All quotes

TOP

  • 4SC1.28 EUR52.38%
  • THERAMETRICS0.08 CHF14.29%
  • WILEX2.96 EUR1.37%

FLOP

  • MOLOGEN5.68 EUR-28.01%
  • MORPHOSYS54.71 EUR-22.45%
  • MEDIGENE11.65 EUR-13.70%

TOP

  • CYTOS1.40 CHF311.8%
  • MEDIGENE11.65 EUR174.1%
  • 4SC1.28 EUR64.1%

FLOP

  • MORPHOSYS54.71 EUR-26.8%
  • WILEX2.96 EUR-13.7%
  • BASILEA106.70 CHF-12.7%

TOP

  • SANTHERA92.00 CHF2259.0%
  • WILEX2.96 EUR393.3%
  • FORMYCON23.15 EUR232.6%

FLOP

  • CYTOS1.40 CHF-54.2%
  • MOLOGEN5.68 EUR-49.9%
  • PAION2.35 EUR-29.6%

No liability assumed, Date: 26.03.2015