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

LicensingSwitzerland

30.08.2016 Swiss biopharma company Ferring has acquired the exclusive development and commercialisation rights to Seikagaku’s Phase III chemonucleolytic leg pain treatment for €85m.

Clinical TrialsFrance

26.08.2016 Adocia said it would discontinue its diabetic foot ulcer programme after its lead product BioChaperone PDGF disappointed in Phase III. The French company will turn the focus on its earlier-stage products.

M&AUKSweden

24.08.2016 Pharma major Pfizer is putting the cherry on top of its multi-billion-dollar takeover of Medivation with the acquisition of AstraZeneca’s antibiotics for a cool US$1.5bn. With the divestment, AZ aims to further sharpen its focus.

FinancingUK

23.08.2016 Cambridge University-allied investor Cambridge Innovation Capital has raised £75m (€87m) to reinvest in technology and healthcare companies. Among the backers were Woodford Investment and Winton Ventures.

R&DDenmark

19.08.2016 Together with partner Johnson & Johnson, Danish biotech Genmab is now one step closer to FDA approval with its combination treatment for multiple myeloma after its submission of a supplemental Biologics License Application. In July, Genmab was granted a Breakthrough Therapy Designation (BTD) for its blood cancer therapy.

ResearchUKEU

17.08.2016 Philip Hammond, the UK’S chancellor has announced the government will guarantee funding for EU-backed "structural and investment fund" projects whose timelines extend beyond the UK’s departure from the EU, provided the projects are signed prior to this year's Autumn Statement on the UK budget. The decision ensures continued funding for farmers, companies and institutions involved in the EU’s Horizon 2020 project. The move could cost the UK government up to £6bn (€6.9bn).

Stock marketsGermany

15.08.2016 Back in April, the American FDA gave the green light for the colorectal cancer-screening test Epi procolon. Now Berlin-based diagnostics specialist Epigenomics is considering a secondary listing in the US. A draft of the required registration document was confidentially submitted to the US Securities and Exchange Commission (SEC).

LicensingDenmark

11.08.2016 Danish Genmab AS has granted Gilead Sciences license to use its DuoBody technology platform to develop bispecific antibodies for the treatment of HIV. The deal is worth up to €252m.

Clinical developmentHungaryIreland

09.08.2016 Hungarian Gedeon Richter and Irish Allergan are staying on course in the face of dissappointing Phase III results for depression drug cariprazine. The partners will now begin another Phase III study needed for approval.

ImmunotherapySpain

08.08.2016 Araclon Biotech’s Alzheimer’s vaccine ABvac40 is going forward into Phase II after showing a good safety profile in Phase I. The promising trial results were presented at the Alzheimer’s Association International Conference.

Quick news provided by GlobeNewswire

View more stock news

Events

All Events

Partner-Events

Current issue

All issues

Product of the week

Products

Stock list

All quotes

TOP

  • ARGEN-X (B)14.20 EUR5.19%
  • MAGFORCE (D)4.71 EUR4.43%
  • DIAMYD MEDICAL -B- (S)7.20 SEK4.35%

FLOP

  • BIONOR PHARMA (N)0.41 NOK-6.82%
  • PHOTOCURE (N)41.00 NOK-6.61%
  • SAREUM HOLDINGS (UK)0.74 GBP-5.13%

TOP

  • NORDIC NANOVECTOR (N)34.50 NOK40.8%
  • KARO BIO (S)43.10 SEK30.6%
  • MOLMED (I)0.42 EUR27.3%

FLOP

  • BIONOR PHARMA (N)0.41 NOK-28.1%
  • GW PHARMACEUTICALS (UK)514.61 GBP-13.6%
  • WILEX (D)1.46 EUR-13.6%

TOP

  • NICOX (F)10.29 EUR441.6%
  • SAREUM HOLDINGS (UK)0.74 GBP236.4%
  • GENMAB (DK)1085.00 DKK83.4%

FLOP

  • BB BIOTECH (D)44.30 EUR-83.1%
  • EVOCUTIS (UK)0.04 GBP-77.8%
  • NEUROVIVE PHARMACEUTICAL AB (S)5.65 SEK-75.4%

No liability assumed, Date: 29.08.2016