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- PMLiVE

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Biomarin

EU nod for BioMarin’s second PKU therapy Palynziq

Palyniq is significant, but gene therapy pipeline most anticipated

- PMLiVE

Orphan drugs market to soar to $224bn by 2024

Cell and gene therapies to fuel growth

- PMLiVE

Lilly to withdraw Lartruvo, a first for accelerated approval failures

Existing patients can use access programme

- PMLiVE

Sobi shows strong growth despite haemophilia competition

Looking to M&A for continued growth

- PMLiVE

Bad news for BMS as Keytruda kidney cancer combo approved

Opdivo and Yervoy combination will be squeezed

- PMLiVE

Gottlieb signs off with flair – and pledge of further drug price commentary

Highlights issues of insulin prices and competition

- PMLiVE

Novo uses voucher for speedy answer on oral semaglutide

Oral version of Ozempic could be diabetes market leader

- PMLiVE

Gottlieb admonishes industry for clinical trial failings

Calls for faster uptake of novel trial design and methods

- PMLiVE

NCI head Sharpless named as interim FDA chief

The search for a permanent replacement is still ongoing

- PMLiVE

Unruffled by ‘outlier’ claims, Vertex CEO upbeat on Orkambi breakthrough

NHS England going public with price makes deal impossible, says Leiden

- PMLiVE

J&J’s Spravato breaks depression drought at FDA

The esketamine nasal spray is the first new-mechanism antidepressant to be licensed in decades

- PMLiVE

FDA chief Scott Gottlieb to step down

Won admiration of industry and public health experts

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