EMA hands Italy's Chiesi a win in its long cam­paign to ad­vance new drugs for rare dis­eases

Five years af­ter Italy’s Chiesi built up its rare dis­ease ef­forts through ac­quir­ing Zymenex, the EMA has come through with a mar­ket­ing OK for a drug ob­tained in that deal.

Lamzede is an en­zyme re­place­ment ther­a­py for the ul­tra-rare al­pha-man­nosi­do­sis, a mul­ti-faceted de­bil­i­tat­ing dis­ease that is es­ti­mat­ed to af­fect 470 peo­ple in the EU. The treat­ment is ap­proved specif­i­cal­ly for non-neu­ro­log­i­cal man­i­fes­ta­tions in pa­tients with mild to mod­er­ate forms of the dis­or­der, a first for this pa­tient pop­u­la­tion.

The dis­ease is caused by a gene mu­ta­tion that leads to se­vere de­fi­cien­cy in al­pha-man­nosi­dase, an en­zyme that breaks down gly­co­pro­teins. As a re­sult, a tox­ic build-up of sug­ars takes place in the body, caus­ing a va­ri­ety of symp­toms from skele­tal ab­nor­mal­i­ties and mus­cle prob­lems to hear­ing loss and mo­tor skill im­pair­ments. Cur­rent treat­ments in­clude bone mar­row trans­plant and pe­riph­er­al blood stem cell trans­plan­ta­tion, in ad­di­tion to oth­er ther­a­pies tar­get­ing in­di­vid­ual symp­toms.

Alessan­dro Chiesi

Des­ig­nat­ed an or­phan drug back in 2005 when it was still in Zymenex’s hands, Chiesi’s Lamzede works by de­liv­er­ing, in­tra­venous­ly, a re­com­bi­nant form of al­pha-man­nosi­dase dubbed vel­manase al­fa to pa­tients. Falling un­der “ex­cep­tion­al cir­cum­stances,” the treat­ment’s ap­proval came af­ter sev­er­al clin­i­cal stud­ies in­volv­ing a to­tal of 34 pa­tients and a pos­i­tive CHMP opin­ion in Jan­u­ary.

The largest of those tri­als, a Phase III, en­rolled 25 pa­tients, and found re­duc­tion in serum oligosac­cha­rides for both the adult and pe­di­atric sub-groups. Those un­der 18 showed pos­i­tive im­prove­ment in func­tion­al tests like the three-minute stair climb­ing test and six-minute walk­ing test, while the adult group achieved sta­bi­liza­tion com­pared to the place­bo arm (who ex­pe­ri­enced more de­cline than the drug arm).

“Lamzede is the first ap­proved treat­ment with dis­ease mod­i­fy­ing po­ten­tial for al­pha-man­nosi­do­sis and there­fore, our next goal is to make this treat­ment avail­able to pa­tients in Eu­rope as soon as pos­si­ble,” said Alessan­dro Chiesi, head of Eu­rope at the Chiesi Group. “The mar­ket­ing au­tho­ri­sa­tion of Lamzede in the EU is a sig­nif­i­cant step­ping stone for pa­tients suf­fer­ing from this dev­as­tat­ing dis­ease and re­in­forces the Chiesi Group’s role in the com­plex and chal­leng­ing world of rare dis­eases.”

Chiesi build­ing in Ger­many. CHIESI DEUTSCH­LAND

Fol­low­ing news of job cuts in Eu­ro­pean R&D ops, Sanofi con­firms it’s of­fer­ing US work­ers an 'ear­ly ex­it'

Ear­li­er in the week we learned that Sanofi was bring­ing out the bud­get ax to trim 466 R&D jobs in Eu­rope, re­tool­ing its ap­proach to car­dio as re­search chief John Reed beefed up their work in can­cer and gene ther­a­pies. And we’re end­ing the week with news that the phar­ma gi­ant has al­so been qui­et­ly re­duc­ing staff in the US, tar­get­ing hun­dreds of jobs as the com­pa­ny push­es vol­un­tary buy­outs with a fo­cus on R&D sup­port ser­vices.

Why would the FDA ap­prove an­oth­er con­tro­ver­sial drug to spur a woman’s li­bido with these da­ta? And why no ex­pert pan­el re­view?

AMAG Pharmaceuticals’ newly approved drug for spurring women’s sexual desire may never make much money, but it’s a big hit at sparking media attention.

The therapy — Vyleesi (bremelanotide) — got the green light from regulators on Friday evening, swiftly lighting up a range of stories around the world, from The New York Times to The Guardian. Several headlines inevitably referred to it as the “female Viagra,” invoking Pfizer’s old erectile dysfunction blockbuster.

But the two drugs have little in common.

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Which top 10 big phar­mas have the most to gain — or lose — over the next 5 years?

When Evaluate Pharma crunched the likely drug sales numbers for the big 10, 2 stood out. 

Takeda, with its big Shire buyout under its belt, is set to almost double its worldwide sales record for 2018 over 5 years, putting it in the big 10 — the 9th spot, to be exact — which is exactly where CEO Christophe Weber wants to be. 

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Roger Perlmutter. Merck via webcast

'Our lega­cy mat­ter­s': Mer­ck maps out Keytru­da king­dom while spot­light­ing ad­vances in vac­cines, hos­pi­tal care

“You can for the mo­ment stop tak­ing notes. You can put down your pens and your pad. I have no slides. I have no sub­stan­tive da­ta. I have no pitch.”

So be­gan Roger Perl­mut­ter’s brief ap­pear­ance on­stage at Mer­ck’s first in­vestor day in five years, where he dived in­to the com­pa­ny’s his­to­ry dat­ing back to 1933. The first em­ploy­ees at Mer­ck Re­search Lab­o­ra­to­ries, hand­picked by founder George W. Mer­ck, were crit­i­cal to Mer­ck’s abil­i­ty to achieve clin­i­cal and com­mer­cial suc­cess.

How small- to mid-sized biotechs can adopt pa­tient cen­tric­i­ty in their on­col­o­gy tri­als

By Lucy Clos­sick Thom­son, Se­nior Di­rec­tor of On­col­o­gy Pro­ject Man­age­ment, Icon

Clin­i­cal tri­als in on­col­o­gy can be cost­ly and chal­leng­ing to man­age. One fac­tor that could re­duce costs and re­duce bar­ri­ers is har­ness­ing the pa­tient voice in tri­al de­sign to help ac­cel­er­ate pa­tient en­roll­ment. Now is the time to adopt pa­tient-cen­tric strate­gies that not on­ly fo­cus on pa­tient needs, but al­so can main­tain cost ef­fi­cien­cy.

J&J's Es­ke­t­a­mine, at cur­rent price, is 'low val­ue for mon­ey' — ICER

For John­son & John­son’s $JNJ phar­ma­ceu­ti­cal ver­sion of the hal­lu­cino­genic anes­thet­ic ke­t­a­mine — es­ke­t­a­mine — to be cost-ef­fec­tive for use in treat­ment-re­sis­tant de­pres­sion in the long term, its list price must be cut by up to half, ICER con­clud­ed in its fi­nal re­port on Thurs­day.

Cog­nizant of the myr­i­ad of ap­proved an­ti­de­pres­sants that of­ten don’t work, the US reg­u­la­tor en­dorsed J&J’s es­ke­t­a­mine, brand­ed as  — Spra­va­to — in March for treat­ment-re­sis­tant de­pres­sion, con­scious that the orig­i­nal cat tran­quil­iz­er is fre­quent­ly used off-la­bel for se­vere de­pres­sion.

In starved an­tibi­ot­ic field, Melin­ta soars as FDA grants speedy drug re­view

Such is the state of af­fairs in an­tibi­ot­ic land that the FDA agree­ing to pri­or­i­ty re­view an ap­pli­ca­tion to ex­pand the use of an an­tibi­ot­ic can rock­et up a stock more than two-fold.

On Wednes­day, Melin­ta Ther­a­peu­tics said its ap­proved an­tibi­ot­ic Baxdela had been grant­ed pri­or­i­ty re­view for use in com­mu­ni­ty-ac­quired bac­te­r­i­al pneu­mo­nia (CAPB). The FDA is ex­pect­ed to make its de­ci­sion by Oc­to­ber 24. Shares of the Con­necti­cut drug­mak­er $ML­NT cat­a­pult­ed, clos­ing up near­ly 224% at $6.41.

Brent Saunders at an Endpoints News event in 2017 — File photo

An­a­lyst call with Al­ler­gan ex­ecs stokes an­tic­i­pa­tion of a plan to split the com­pa­ny in ‘a month or two’

So what’s up at Al­ler­gan?

Ear­li­er this week the ubiq­ui­tous Ever­core ISI an­a­lyst Umer Raf­fat was on the line with com­pa­ny ex­ec­u­tives to probe in­to the lat­est on the num­bers as well as CEO Brent Saun­ders’ re­cent de­c­la­ra­tion that he’d be do­ing some­thing de­fin­i­tive to help long-suf­fer­ing in­vestors who have watched their shares dwin­dle in val­ue.

He came away with the im­pres­sion that a sig­nif­i­cant com­pa­ny split is on the way. And not on some dis­tant time hori­zon.

John Reed at JPM 2019. Jeff Rumans for Endpoints News

Sanofi's John Reed con­tin­ues to re­or­ga­nize R&D, cut­ting 466 jobs while boost­ing can­cer, gene ther­a­py re­search

The R&D reorganization inside Sanofi is continuing, more than a year after the pharma giant brought in John Reed to head the research arm of the Paris-based company.

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