Gottlieb sets a high bar for his successor
Scott Gottlieb's two-year tenure as commissioner of the FDA will end next month following his surprise resignation on Tuesday.
His appointment has been one of the most successful of a high ranking government official under the Trump administration, raising industry concerns about who will replace him.
The pharma industry has lost a proactive commissioner whose manifesto has strongly emphasised supporting innovation. A record number of novel drug approvals followed, including the first CAR-T and gene therapies, and the first cancer treatments to be approved for biomarker specific tumours regardless of histology.
Analysis - ViewPoints: Gottlieb begins his long goodbye
FDA data also show that the number of new drugs approved on the basis of surrogate endpoints has increased significantly since 2017; a trend supported in part by industry's considerable investment in oncology R&D and the approval of numerous new cancer therapies on the basis of smaller, single-arm studies.
Gottlieb has also played a key role in raising the FDA's profile as a more active player in driving the discussion on drug pricing and ways to lower the cost of medicines for patients, not to mention leading aggressive public health campaigns targeting the opioid epidemic, the tobacco industry (in particular related to e-cigarettes) and encouraging the use of vaccines.
The question now is whether his successor will take hold of the baton and run with it.
The FDA confirmed this week approval of Johnson & Johnson's Spravato for treatment-resistant depression. Spravato, which is related to ketamine, is the first novel major depression therapy to be approved in decades. It is administered via a nasal spray and works faster than currently available depression medicines.
The number of patients who don't respond to existing depression therapies is significant (approximately a third of people with major depression have failed at least two therapies), but this won't guarantee that Spravato is a rapid-fire blockbuster for Johnson & Johnson, despite the fact it works by way of a novel mechanism of action
For a start, it didn't succeed in two pivotal-stage studies (of the five run), which has typically been the regulatory benchmark for new depression drugs and will not be an easy medication to administer. For example, patients will require monitoring for two hours after they receive treatment.
Our recent snap-poll of 100 psychiatrists provides an idea on how Spravato may be used, with around half of respondents suggesting they are 'eager' or 'very eager' to use the drug; enthusiasm was higher among the small proportion of physicians who have used ketamine off-label to treat depression.
Physicians also appear to find positive data for Spravato - from one pivotal study and one withdrawal, or maintenance, study - relatively compelling, though to a similar extent they recognise that logistical challenges will have some impact in limiting uptake.
Illustrating how challenging it has been to bring new depression therapies to market, Allergan confirmed this week that its experimental drug rapastinel failed to hit its primary endpoint in a handful of pivotal stage studies.
More musical chairs at Gilead
Investors in Gilead Sciences looking for reassurances about the company's future strategy will be pleased that Daniel O'Day is now installed as CEO. The appointment of O'Day - who previously headed up Roche's pharmaceuticals business - appears to clearly signal Gilead's intent to further expand its footprint in oncology.
To what extent this focus will sharpen on cell therapy has been a point of discussion since O'Day was announced as successor to John Milligan back in December, however. In contrast to Gilead's 2017 acquisition of Kite Pharma for $11.9 billion, Roche (and O'Day) have lauded the science behind CAR-T, but questioned its commercial applicability, choosing to focus on other treatment approaches, such as bispecifics instead.
Confirmation on Tuesday that oncology head Alessandro Riva, who was influential in the acquisition of Kite, will leave Gilead at the end of the month after just two years will only further speculation about the company's future investment in cell-based therapies.
Big Pharma's interest in gene therapies shows no sign of cooling
In announcing a deal to acquire Nightstar Therapeutics for $800 million this week, Biogen has retained buyer’s momentum in the gene therapy space.
Whether the deal is sufficient to appease investors growing increasingly concerned that Biogen needs to diversify its pipeline is another matter, however.
SEC documents published on Thursday also reveal that Roche's recently announced acquisition of Spark Therapeutics for $4.3 billion saw the Swiss company outmanoeuvre two competing bidders to secure its target.
FirstWord from the frontlines
A leading key opinion leader told FirstWord that improvements in predictive modelling tools to pre-diagnose rapidly progression osteoarthritis (RPOA) will be critical to maximise the commercial opportunity for anti-NGF monoclonal antibodies being developed for pain indications.
In another interview, a leading NASH expert described Intercept's Ocaliva as approvable in NASH, but questioned its therapeutic window and suggested the bar has been set relatively low for future treatment options.
Despite the 10mg version of Xeljanz linked to increased risk of pulmonary embolism not being approved for RA, rheumatologists do expect some detrimental impact on use, particularly in Europe - see Physician Views Results: Position of Xeljanz in rheumatoid arthritis could come under some pressure, particularly in the less established European market
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