BMS’ Opdivo gets ‘breakthrough’ badge for bladder cancer
28 June 2016

US regulators have awarded Bristol-Myers Squibb's immunotherapy Opdivo its sixth breakthrough therapy status, this time for its potential in treating bladder cancer.

X-Linked Hypophosphatemia Drug Gets FDA Breakthrough Status
28 June 2016

The FDA granted a Breakthrough Therapy Designation for Ultragenyx’s orphan drug – KRN23—to treat children with X-linked hypophosphatemia (XLH).

Cannabinoid Product Shows Positive Phase 3 Trial Results
27 June 2016

Last week, we reported GW Pharmaceuticals announced a 4th orphan indication for Epidiolex – infantile spasms – will be studied in a pivotal Phase 3 study beginning in the 4th quarter of 2016.

EU nod for Gilead’s single-pill HIV therapy
24 June 2016

European regulators have granted marketing authorisation to Gilead's Odefsey for the treatment of certain patients with HIV-1 infection.

Cost regulators are sticking with a decision to bar some patients with acute myeloid leukaemia from routine NHS access to Celgene's Vidaza
24 June 2016

The National Institute for Health and Care Excellence has rejected the drug's use as an option to treat AML in patients aged 65 years or older with more than 30 percent bone marrow blasts who are

Jakafi Good for Acute Graft-vs-Host Disease?
23 June 2016

Jakafi (ruxolitinib) is going after another indication –this time to treat patients with acute graft-vs-host disease.

Within the next three months patients with lupus should get 'routine' access to a new treatment option on the NHS in England and Wales after cost regulators issued final guidance for GlaxoSmithKline's Benlysta
23 June 2016

In line with draft recommendations, the National Institute for Health and Care Excellence has recommended use of Benlysta (belimumab) to treat systemic lupus erythematosus (SLE), but only under a m

Characteristics of patients contacting a center for undiagnosed and rare diseases
21 June 2016

Little is known about the characteristics of patients seeking help from dedicated centers for undiagnosed and rare diseases.