FDA an­a­lysts picked apart the da­ta, high­light­ed an ab­sence of over­all sur­vival ben­e­fit and not­ed that As­traZeneca had yet to hit the mark re­quired for ear­li­er pan­cre­at­ic can­cer drug ap­provals — but it didn’t make any dif­fer­ence in the end. Short­ly af­ter a slight ma­jor­i­ty of ex­pert ad­vis­ers vot­ed 7 to 5 in fa­vor of us­ing Lyn­parza as a main­te­nance ther­a­py for germline BR­CA-mu­tat­ed metasta­t­ic ade­no­car­ci­no­ma of the pan­creas af­ter 16 weeks of dor­man­cy, the FDA fol­lowed up with a for­mal sanc­tion of its own on Mon­day.

Dave Fredrick­son

The move fur­ther so­lid­i­fies the lead­ing po­si­tion As­traZeneca and Mer­ck hold in the PARP field as Lyn­parza con­tin­ues to rack up new suc­cess­es in on­col­o­gy. This new OK fol­lows land­mark ap­provals in ovar­i­an and breast can­cer as the ma­jor league part­ners line up new fields to con­quer with a drug that tar­gets a re­pair path­way for cells.

The reg­u­la­to­ry bar is set par­tic­u­lar­ly low in on­col­o­gy, and that’s es­pe­cial­ly true for hard-to-treat cas­es like pan­cre­at­ic can­cer, where sur­vival ben­e­fits are low. The ap­proval was large­ly based on da­ta in­di­cat­ing a longer span of pro­gres­sion-free sur­vival for pa­tients, though reg­u­la­tors not­ed is­sues with the tri­al used to gain the OK. But Lyn­parza is well known to the FDA, mak­ing it eas­i­er to take a fly­er on it.

Dave Fredrick­son, As­traZeneca’s EVP of the on­col­o­gy busi­ness unit, said:

Pa­tients with ad­vanced pan­cre­at­ic can­cer his­tor­i­cal­ly have faced poor out­comes due to the ag­gres­sive na­ture of the dis­ease and lim­it­ed treat­ment ad­vances over the last few decades. Lyn­parza is now the on­ly ap­proved tar­get­ed med­i­cine in bio­mark­er-se­lect­ed pa­tients with ad­vanced pan­cre­at­ic can­cer.

Fewer Approvals, but Neurology Rivals Oncology and Sees Major Innovations

This report, the fourth in our Trinity Drug Index series, outlines key themes and emerging trends in the industry as we progress towards a new world of targeted and innovative products. It provides a comprehensive evaluation of the performance of novel drugs approved by the FDA in 2016, scoring each on its commercial performance, therapeutic value, and R&D investment (Table 1: Drug ranking – Ratings on a 1-5 scale).

For start-up biotechnology companies and resource stretched pharmaceutical organisations, launching a novel product can be challenging. Lean teams can make setting a launch strategy and achieving your commercial goals seem like a colossal undertaking, but can these barriers be transformed into opportunities that work to your brand’s advantage?
We spoke to Managing Consultant Frances Hendry to find out how Blue Latitude Health partnered with a fledgling subsidiary of a pharmaceutical organisation to launch an innovative product in a
complex market.
What does the launch environment look like for this product?
FH: We started working on the product at Phase II and now we’re going into Phase III trials. There is a significant unmet need in this disease area, and everyone is excited about the launch. However, the organisation is still evolving and the team is quite small – naturally this causes a little turbulence.

→ Less than a year ago Gilead’s antiviral remdesivir failed to make the cut as investigators considered a raft of potential drugs that could be used against an Ebola outbreak. But it may gain a new mission with the outbreak of the coronavirus in China, which is popping up now around the world.

Gilead put out a statement saying that they’re now in discussions with health officials in the US and China about testing their NUC against the virus. It’s the latest in a growing lineup of biopharma companies that are marshaling R&D forces to see if they can come up with a vaccine or therapy to blunt the spread of the virus, which has now sickened hundreds, killed at least 17 people and led the Chinese government to start quarantining cities.

Back at the beginning of 2015, Deerfield Management co-led a $10 million Series C for a private gene therapy startup, reshaping the company and bringing in new leaders to pave way for an IPO just a year later.

Fast forward four more years and the startup, AveXis, is now a subsidiary of Novartis marketing the second-ever gene therapy to be approved in the US.

For its part, Deerfield has also grown more comfortable and ambitious about the nascent field. And the investment firm is now putting down its biggest bet yet: a $1.1 billion contract development and manufacturing facility to produce everything one needs for cell and gene therapy — faster and better than how it’s currently done.

Different therapeutic traits brandished by the three approved therapies for sickle cell disease all extend life expectancy, but their impact on quality of life is uncertain and their long-term cost-effectiveness is not up to scratch according to the thresholds considered reasonable by ICER, the non-profit concluded in a draft guidance report on Thursday.

Sickle cell disease (SCD), which encompasses a group of inherited red blood cell disorders that typically afflict those of African ancestry, impacts hemoglobin — and is characterized by episodes of searing pain as well as organ damage.