Friday Five – the pharma week in review

Gene therapies pose a pricing dilemma; Alnylam finds the commercial game tough; Can democrats and republicans meet in the middle on drug prices? Expect more pharma innovation from China, and fast, say experts; Sage on the verge of a therapeutic breakthrough

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Novartis tells payers to play the long game

A new one-time gene therapy for spinal muscular atrophy set to be approved by regulators next year would be cost effective if sold at between $4 million and $5 million a dose, Novartis said this week.

The Swiss company will price AVXS-101 lower than this, but payers must take a more considered view of the long-term cost benefits of transformational medicines, suggested CEO Vas Narasimhan.

Analysts at Vontobel said Novartis' R&D day in London showcased the company's pivot to becoming a fully integrated specialised medicines company is now "in full swing."

Biosimilars are an equally disruptive strategy in Novartis' strategic playbook, the head of its Sandoz division Richard Francis said at an industry conference on Monday.

By increasing access to current standards of care at a lower cost and potentially reducing expenditure, greater use of biosimilars will free up resources for innovative therapies, he said.

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A tough start for Alnylam and RNAi

Big Pharma can better afford transformative therapies to be adopted slowly if that's what it takes to get payers on side; new commercial-stage biotech's less so.

Earlier this year, Alnylam Pharmaceuticals delivered an important clinical and regulatory breakthrough when it secured US approval of Onpattro; a treatment for hereditary transthyretin-mediated amyloidosis (hATTR), which became the first marketed RNAi therapeutic.

Third-quarter results reported on Wednesday showed, however, that Onpattro has so far failed to meet early commercial expectations. Sales of just $0.5 million were below consensus forecasts of $4.2 million.

Alnylam stressed that it is early days, but the performance of Onpattro to date provides further evidence that even the most significant scientific breakthroughs don't translate to immediate market success - more here.

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After the mid-terms, where next for US drug pricing?

Where drug pricing is concerned, President Donald Trump has said a lot, but done relatively little.

His administration's latest proposals include international referencing pricing as a means to bring down expenditure under Medicare Part B; democrats want to go further, ideally allowing all of Medicare to negotiate drug prices directly.

Any progress will now require bipartisan support and both sides have suggested there could be common ground. Analysts were quick to suggest, however, that Democratic control of the house and Republican control of the senate may ultimately represent a 'best case' outcome for drugmakers, describing the latent threat of increased pressure on drug prices as 'headline risk' at worst.

Watch this space.

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China on the verge of becoming a global pharma innovation leader, say experts

Delegates attending the Financial Times Global Pharmaceutical and Biotechnology conference in London this week were told by experts that the prediction Chinese innovation in drug development will make a global impact by 2025 is conservative.

Christian Hogg, CEO of Chi-Med, was quick to stress that China's rapid emergence on the world pharma stage is no overnight success story, but instead has taken two decades "of very hard work by very clever people." He concedes, however, that a more recent overhaul of the regulatory system, reform of the drug reimbursement process and the level of government investment in healthcare has been extraordinary. 

Big Pharma (as we know it) must increasingly view China as both a significant opportunity and threat it seems.

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Sage passes/Alkermes fails at FDA AdCom

Near unanimous support from an FDA AdCom panel in favour of approving Sage Therapeutics' post-partum depression treatment Zulresso should provide much needed momentum in this field - more here

A day earlier, the same panel of experts had shot down Alkermes depression candidate ALKS 5461 by largely agreeing that available clinical data did not support approval of the opioid-based candidate.

Developing new depression drugs has become notoriously hard. Sage told FirstWord earlier this year it thinks it has found an effective new approach. Its initial interactions with the FDA suggest it could be onto something.

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