May 24 is go­ing to go down as a big day in No­var­tis’ 2019 high­light reel.

Not on­ly did the FDA stamp an OK on the prospec­tive gene ther­a­py block­buster Zol­gens­ma, with a record-set­ting $2.1 mil­lion price tag, reg­u­la­tors al­so green-light­ed alpelis­ib, one of their oth­er top block­buster can­di­dates of the year.

Vas Narasimhan Linkedin

These are 2 of the slate of 4 block­busters the phar­ma gi­ant $NVS high­light­ed for a com­mer­cial roll­out this year. And it’s the third big ap­proval this year, be­hind the MS drug Mayzent, mak­ing 2019 a wa­ter­shed year for the com­pa­ny and new CEO Vas Narasimhan.

Alpelis­ib, ini­tial­ly code-named BYL719, will now hit the mar­ket as Piqray. It will be sold for HR+/HER2- ad­vanced breast can­cer with a PIK3CA mu­ta­tion — the first such tar­get­ed ther­a­py OK’d by the agency.

The da­ta cleared all the hur­dles need­ed for the FDA. Piqray plus ful­ves­trant near­ly dou­bled me­di­an pro­gres­sion-free sur­vival com­pared to ful­ves­trant alone in HR+/HER2- ad­vanced breast can­cer pa­tients with a PIK3CA mu­ta­tion (me­di­an PFS 11.0 months vs 5.7 months; HR=0.65). The ORR for the com­bo was 35.7% vs 16.2% for ful­ves­trant alone, p=0.0002.

It won’t come cheap. The drug will be sold at a whole­sale ac­qui­si­tion price of $15,500 a month, or right around $200,000 a year.

That’s a win­ner. It’s al­so a source of pride for No­var­tis R&D. The multi­na­tion­al bought Zol­gens­ma a year ago when it was well on its way, but they brought this drug all the way up from their dis­cov­ery group at NI­BR, mak­ing it a home­grown suc­cess sto­ry. And the FDA ex­e­cut­ed the ap­proval as the first un­der its re­al-time re­view pro­gram, which lets reg­u­la­tors start con­sid­er­ing an ap­proval be­fore the for­mal sub­mis­sion of the ap­pli­ca­tion.

More amaz­ing news. Thrilled to share this mile­stone ap­proval of our new tar­get­ed ther­a­py. More than ten years in the mak­ing, this pro­gram was sup­port­ed by count­less in­ter­nal NI­BR cham­pi­ons. Con­grats to all who car­ried this ther­a­py through to pa­tients. https://t.co/fb­Jt­DRqITx

— Jay Brad­ner, M.D. (@jay­brad­ner) May 24, 2019

“To­day’s ap­proval is ex­pect­ed to change the way we prac­tice med­i­cine in ad­vanced breast can­cer. For the first time, physi­cians can test for PIK3CA bio­mark­ers and de­vel­op a treat­ment plan based on the ge­nom­ic pro­file of a pa­tient’s can­cer,” said Fab­rice An­dré, a pro­fes­sor in the de­part­ment of med­ical on­col­o­gy at In­sti­tut Gus­tave Roussy in Ville­juif, France, and the lead in­ves­ti­ga­tor in the study.

No­var­tis is fresh off mak­ing some bold promis­es to an­a­lysts about the 25-plus prospec­tive drugs it has in the pipeline. That gives the still new CEO Narasimhan a sol­id foun­da­tion to build his com­pa­ny on new drug cam­paigns, with the in-house de­vel­op­ment work sup­ple­ment­ed by a slate of planned bolt-on deals.

Medical animation has in recent years become an increasingly important tool for conveying niche information to a varied audience, particularly to those audiences without expertise in the specialist area. Science programmes today, for example, have moved from the piece-to-camera of the university professor explaining how a complex disease mechanism works, to actually showing the viewer first-hand what it might look like to shrink ourselves down to the size of an ant’s foot, and travel inside the human body to witness these processes in action. Effectively communicating a complex disease pathophysiology, or the novel mechanism of action of a new drug, can be complex. This is especially difficult when the audience domain knowledge is limited or non-existent. Medical animation can help with this communication challenge in several ways.
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As a potentially powerful flu season looms, so does a big test for Roche and its new flu drug, Xofluza. The Swiss giant just got a small boost in advance of that test as the FDA expanded Xofluza’s indication to include patients at high risk of developing flu-related complications.

Xofluza (baloxavir marboxil) was approved last October in the US, the first landmark flu drug approval in 20 years and a much-needed green light for a company that had watched its leading flu drug Tamiflu get eaten alive by generics. Like its predecessor, the pill offered a reduction in flu symptoms but not a cure.

The first-ever Ebola vaccine is on the precipice of approval after the European Medicine’s Agency (EMA) backed the Merck product in this week’s roster of recommendations.

The drugmaker $MRK began developing the vaccine, christened Ervebo, during the West African outbreak that occurred between 2014 and 2016, killing more than 11,000.

The current outbreak in the Democratic Republic of Congo (DRC) has shown case fatality rates of approximately 67%, the agency estimated. Earlier this year, the WHO declared the outbreak — which so far has infected more than 3,000 people — a public health emergency of international concern.