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rare disease

This page shows the latest rare disease news and features for those working in and with pharma, biotech and healthcare.

Shire rebuffs Takeda bids, as Allergan bows out

Shire rebuffs Takeda bids, as Allergan bows out

Rare disease specialist Shire issued its own statement, noting that Takeda had made three approaches, starting with a £41bn overture on 29 March and followed by a £43bn bid on 11

Latest news

  • Rigel and Ultragenyx claim approvals for rare drugs Rigel and Ultragenyx claim approvals for rare drugs

    The FDA gave the go-ahead to a pair of rare disease therapies this week, both marking the first-ever approvals for developers Rigel Pharmaceuticals and Ultragenyx. ... patients. He’s predicting sales could reach $336m in 2022. Ultragenyx has also

  • P&G agrees $4.2bn buy of Merck KGaA’s consumer health unit P&G agrees $4.2bn buy of Merck KGaA’s consumer health unit

    In the last few weeks Sanofi has sold its European generics business to Advent, GlaxoSmithKline divested its rare disease unit to start-up Orchard Therapeutics after buying Novartis out of a

  • Sanofi sells European generics to Advent for $2.4bn Sanofi sells European generics to Advent for $2.4bn

    The restructuring has already seen it bolster its pipeline in rare diseases with the acquisitions of Bioverativ for $11.6bn and Ablynx for 3.9bn after missing out on earlier bids ... Just this month, for example, GlaxoSmithKline divested its rare disease

  • GSK hands rare disease assets over to Orchard GSK hands rare disease assets over to Orchard

    GSK hands rare disease assets over to Orchard. The UK pharma group will take a 19.9% stake in its ex-rival. ... GlaxoSmithKline has decided the future of its rare disease business - and it lies outside the company.

  • Alexion adds to pipeline rebuild with $855m Wilson buy Alexion adds to pipeline rebuild with $855m Wilson buy

    Alexion adds to pipeline rebuild with $855m Wilson buy. Also acquires the Swedish group’s lead product for Wilson’s disease. ... Alexion Pharma’s drive to reduce its reliance on cash cow Soliris has gone up a gear with a $855m offer for rare

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Latest Intelligence

  • Accelerating diagnosis in rare disease Accelerating diagnosis in rare disease

    Accelerating diagnosis in rare disease. Timely diagnosis in rare disease is one of the fundamental unmet needs for patients, healthcare professionals (HCPs) and biopharma. ... However, as referring and prescribing doctors in rare disease can be small in

  • Accelerating diagnosis in rare disease Accelerating diagnosis in rare disease

    Accelerating diagnosis in rare disease. Timely diagnosis in rare disease is one of the fundamental unmet needs for patients, healthcare professionals (HCPs) and biopharma. ... However, as referring and prescribing doctors in rare disease can be small in

  • Significant figures Significant figures

    ALIS (Amikacin liposome inhalation suspension) is being developed as a treatment for refractory non tuberculous Mycobacterial (NTM) lung disease, a rare progressive, destructive infection caused by mycobacterium avium complex. ... NTM is a rare

  • Deal Watch January 2018

    Rare blood disease products in vogue - Sanofi part 2. In the early part of January it was made public that Novo Nordisk had made an unsolicited $3.1bn (28 per share) ... Sanofi was also interested in caplacizumab, which fits not only with its rare

  • The real-world data conundrum The real-world data conundrum

    promise around treating rare diseases. ... However, there is still high unmet need: fewer than one in ten patients with a rare condition receive disease-specific treatment and approved medicines are available for only 5% of rare

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Latest appointments

  • Abeona Therapeutics names CEO Abeona Therapeutics names CEO

    Rare genetic disease focused biopharma Abeona Therapeutics has named its chief executive officer in the form of Carsten Thiel. ... Steven Rouhandeh, executive chairman of Abeona, said: “As CEO, Carsten Thiel brings a unique combination of extensive

  • Therachon poaches Shire’s Aled Williams Therachon poaches Shire’s Aled Williams

    Williams has more than 25 years’experience in healthcare and pharmaceuticals and most recently he served as vice president and global strategy head for rare gastrointestinal diseases at Shire. ... Commenting on the recent appointment, Luca Santarelli,

  • Open Health creates new role Open Health creates new role

    Open Health creates new role. Gavin Jones joins the agency as its director of rare disease. ... In order to strengthen their experience and expertise, healthcare communications and market access group Open Health has appointed Gavin Jones into its newly

  • Mereo BioPharma appoints head of patient access and commercial planning Mereo BioPharma appoints head of patient access and commercial planning

    rare disease commercial infrastructure. ... In this role, Hughes-Wilson was responsible for Sobi’s commercialisation approach and led pricing, reimbursement and access teams for the group’s rare disease product portfolio.

  • Amryt Pharma appoints new regulatory affairs head Amryt Pharma appoints new regulatory affairs head

    Amryt Pharma appoints new regulatory affairs head. Derval O’Carroll joins the rare disease-focused biopharma from Retrophin. ... Prior to joining Amryt, she served as senior director of regulatory affairs for rare disease group Retrophin.

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