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21.01.2013 - The EMA has withdrawn an MAA of Switzerland’s Santhera for Raxone, a synthetic coenzyme Q10 derivative against hereditary blindness.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

PoliticsEU

15.09.2014 Jean Claude Juncker, the European Commission President-elect revealed his new Commissioners last week, whilst announcing a change in the units dealing with medicines, medical devices and health technology.

BiosimilarsGermanyFrance

12.09.2014 The 2011 collaboration of Boehringer Ingelheim and Eli Lilly is bearing fruit. An insulin biosimilar developed through the partnership has now been approved in Europe - the first-ever generic version of this class.

FinancingNetherlands

11.09.2014 Cystic fibrosis specialist Proqr Therapeutics has set the terms for its upcoming IPO at US-American stock exchange NASDAQ.

Clinical TrialsUKSweden

09.09.2014 In the midst of the hustle and bustle of the European Respiratory Society’s International Congress 2014, Astrazeneca quietly announced the disappointing results of its latest benralizumab study.

BusinessDenmark

04.09.2014 After their arthritis product’s Phase IIb failure, Novo Nordisk A/S is pulling out of all research and development activities within inflammatory disorders.

BusinessBelgiumIrelandSwitzerland

02.09.2014 In the face of declining revenue, Belgian biopharmaceutical company Thrombogenics has decided to spin out its cancer research and development activities.

ResearchUKSwitzerlandItaly

29.08.2014 Just a week ago, Glaxosmithkline defused the WHO’s claim that an Ebola vaccine could be ready by 2015. Now, the British company’s own candidate vaccine is to be rushed to human trials with the help of funding from an international consortium.

BusinessGermany

24.08.2014 Germany's Morphosys is licensing a preclinical bi-specific antibody for the treatment of prostate cancer from Emergent Biosolutions. The US developer will rake in up to €138m for the commercialisation rights to the compound.

BusinessUK

20.08.2014 Biopharmaceutical contract manufacturer Fujifilm Diosynth Biotechnologies is expanding its cell culture manufacturing capacity further with a new 2,000l single-use bioreactor at the company’s site in Billingham, UK.

Events

All Events

Current issue

All issues

Product of the week

Products

Stock list

All quotes

TOP

  • CO.DON2.84 EUR4.80%
  • VITA 343.98 EUR4.19%
  • MEDIGENE4.07 EUR2.26%

FLOP

  • THERAMETRICS0.08 CHF-11.11%
  • CYTOS0.24 CHF-4.00%
  • BASILEA97.25 CHF-1.22%

TOP

  • SANTHERA93.80 CHF46.3%
  • CO.DON2.84 EUR19.3%
  • FORMYCON8.22 EUR18.8%

FLOP

  • EVOTEC3.09 EUR-17.6%
  • MEDIGENE4.07 EUR-13.8%
  • 4SC1.16 EUR-12.8%

TOP

  • SANTHERA93.80 CHF2036.7%
  • CO.DON2.84 EUR226.4%
  • PAION2.69 EUR158.7%

FLOP

  • CYTOS0.24 CHF-94.0%
  • THERAMETRICS0.08 CHF-46.7%
  • MERCK KGAA67.56 EUR-41.3%

No liability assumed, Date: 15.09.2014