Novartis’ Zolgensma controversy drags on
In response to an FDA inspection report, Novartis has said former AveXis chief scientific officer Brian Kaspar and senior vice president of R&D Allan Kaspar are to blame for manipulated data submitted to the FDA as part of the regulatory application for Zolgensma. See - ViewPoints: Novartis looks to build the case for Zolgensma's durability.
Inconsistences in early-stage mice data for the spinal muscular atrophy (SMA) gene therapy first came to public attention early last month. Novartis has come under FDA scrutiny for its delay in disclosing these inconsistences to the agency and opening up a formal probe into the matter.
The FDA has previously signalled there is no reason to question the efficacy or safety of Zolgensma based on these findings and a number of key opinion leaders (KOLs) recently interviewed by FirstWord for a forthcoming SMA report said these data inconsistences are unlikely to materially affect use of the product, which was approved by the FDA in May.
How quickly Zolgensma - which costs $2.1 million as a ‘one-time’ therapy, will be adopted remains unclear, however. Evidence of a durable effect will be essential but could prove challenging due to real world considerations, argue some experts.
Vitrakvi approved in Europe
Bayer scored notable progress for its R&D pipeline this week with the cancer drug Vitrakvi approved for use in Europe.
Notably it is the first tumour agnostic cancer therapy to be approved in the EU, indicated for the treatment of adult and paediatric patients with solid tumours that display an NTRK gene fusion. Vitrakvi has impressed in pivotal-stage studies - with more data due to be presented at ESMO this weekend - but could provide commercial challenges due to the rarity of the genetic signature it targets. An ongoing partnership with Foundation Medicine to develop blood-based gene sequencing diagnostics could be key to unlocking Vitrakvi’s potential in the long term.
Bayer told FirstWord “it is our intent that no eligible patient with TRK fusion cancer will go without this highly effective therapy. Bayer will work together with local government agencies and payers to facilitate patient access on the local level, according to the value the product offers and the contribution towards reducing the burden of a disease for society.”
Pricing is likely to be on Bayer’s agenda. In the US - where Vitrakvi was approved last year - Roche has priced its similar drug Rozlytrek at nearly half the cost in a bid to drive faster adoption.
Rybelsus off and running
The FDA gave Novo Nordisk the long-awaited thumbs up to begin marketing Rybelsus (semaglutide), meaning the all-important question now becomes whether the oral GLP-1 agonist will be a revolutionary breakthrough or evolutionary step forward in the management of type 2 diabetes.
Novo Nordisk’s task now is to position Rybelsus further upstream than the third-line setting where injectable GLP-1 drugs like Eli Lilly’s Trulicity (dulaglutide) and its own Ozempic (semaglutide) are typically prescribed. Hoping to perform a delicate balancing act, the company set Rybelsus’ list price at parity the injectables, which analysts believe is just the opening salvo in protracted negotiations that will dictate where it appears on formularies and in turn determine when and how often the drug is used. See – ViewPoints: Biggest question on Novo’s Rybelsus answered, now for the important ones.
Also stay tuned early next week for – KOL Views: Gauging the impact of the long-awaited arrival of Novo Nordisk’s Rybelsus
Previewing a PARP showdown
UK pharma rivals AstraZeneca and GlaxoSmithKline face a showdown at ESMO on Saturday, when data is presented for their respective PARP inhibitors Lynparza and Zejula as first-line maintenance therapy for all comer ovarian cancer patients.
In the PAOLA-1 study, Lynparza is being evaluated in combination with Avastin – an approval that AstraZeneca and partner Merck & Co. hope will deliver synergistic benefits. By comparison, Zejula has been studied as a monotherapy in this setting in the PRIMA study.
Analysts at Credit Suisse suggested this week that investors have become increasingly bullish towards the combination strategy favoured by AstraZeneca and Merck, but note nuances in study design make cross-trial comparisons challenging; for example, PRIMA has enrolled a sicker baseline patient group versus PRIMA, they said.
Intriguingly, data from PAOLO-1 will be included in part of the ESMO press programme but results from PRIMA not, adding further speculation before results are presented in full on Saturday afternoon. GlaxoSmithKline arguably has more riding on the outcome of these data than its UK rival; strong data will help to justify the $5 billion it spent to acquire Tesaro late last year. See – Spotlight On: What to watch at ESMO 2019.
US pricing reform teeters in the balance
Some rare signs of bipartisan cooperation to take on drug pricing in the US emerged after US House Majority Leader Nancy Pelosi introduced a bill earlier this month to take an aggressive stance on drug pricing; Republicans concerned about maintaining their Senate majority seemed unusually eager to work with Democrats to advance the bill, complimented by a separate piece of legislation advanced from the US Senate Finance Committee in July.
However, the Senate bill didn't make it out of committee without some challenges of its own, with debate festering over the legislation's proposal to cap price increases in Medicare Part B and Part D, as well as the use of international reference pricing- ultimately leading to its unanimous approval from Democrats on the committee, with a divided vote from Republicans. New developments last week suggest that the odds of the bill's success may now be even longer, with the proposal stuck in purgatory after reports that Senate majority leader Mitch McConnell will decline to bring the bill to a floor vote this year- possibly tied to an impeachment proceeding against President Donald Trump that is likely to dominate headlines for weeks.
The Senate proposal is uniformly less ambitious than its House version, giving it sustainably better odds of moving forward- albeit still limited by fractious Republican support, and with its prospects now dimmed as the Senate works through more urgent matters.
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