More re­search is need­ed to jus­ti­fy use of 'med­i­c­i­nal' cannabis: UK agen­cies

A con­cert­ed cam­paign to un­lock the use of cannabis to treat se­vere child­hood epilep­sy prompt­ed the British au­thor­i­ties last year to sanc­tion its use in cer­tain pa­tients, when oth­er med­i­cines have failed, af­ter con­sul­ta­tion with a spe­cial­ist doc­tor. On Thurs­day, NHS Eng­land and cost-ef­fec­tive­ness watch­dog NICE un­veiled their pre­lim­i­nary rec­om­men­da­tions on the adop­tion of cannabis-de­rived med­i­cines for a va­ri­ety of con­di­tions. In short, they want more re­search to jus­ti­fy the med­ical use of cannabis.

NHS Eng­land is­sued a re­view that un­der­scored a pauci­ty of long-term safe­ty and ef­fi­ca­cy of med­i­c­i­nal cannabis, ac­knowl­edg­ing that clin­i­cians have found that some pa­tients with se­vere treat­ment-re­sis­tant epilep­sy do re­spond to cannabis-based ther­a­py and that ob­ser­va­tion­al da­ta sug­gest cannabis ther­a­py can di­min­ish seizure fre­quen­cy, re­sult­ing in few­er trips to emer­gency rooms and a bet­ter qual­i­ty of life.

While med­i­c­i­nal cannabis was be­ing con­sid­ered on a case-by-case ba­sis since last No­vem­ber, the lack of ran­dom­ized con­trol tri­al (RCT) da­ta is a ma­jor hur­dle to pre­scrib­ing, in ad­di­tion to the cost of ac­quir­ing the prod­uct, which is not read­i­ly at­tain­able on the NHS or by NHS Trusts.

Con­se­quent­ly, the NHS rec­om­mend­ed two clin­i­cal tri­als be set up.

In par­al­lel, NICE rec­om­mend­ed fur­ther re­search in­to the use of cannabis-based prod­ucts in in­di­ca­tions such as fi­bromyal­gia, chron­ic pain, nau­sea and vom­it­ing and spas­tic­i­ty.

The agency al­so high­light­ed that it was un­able to make a rec­om­men­da­tion for the use of cannabis-de­rived med­i­cines in se­vere treat­ment-re­sis­tant epilep­sy, cit­ing a lack of clear ev­i­dence of ben­e­fit. (GW Phar­ma’s FDA-ap­proved cannabis-de­rived drug for epilep­sy, Epid­i­olex, is un­der Eu­ro­pean re­view.)

Si­mon Wig­glesworth Epilep­sy Ac­tion

“For those af­fect­ed by se­vere epilep­sies, hope is of­ten in short sup­ply. We recog­nise how dis­ap­point­ing the draft rec­om­men­da­tions will be for many. Hav­ing al­ready wait­ed al­most a year for this guid­ance, the prospect of wait­ing many years more for tri­als to be com­plet­ed is un­ac­cept­able,” Si­mon Wig­glesworth, deputy chief ex­ec­u­tive at UK-based Epilep­sy Ac­tion, told End­points News.

On the ba­sis of a lack of cost-ef­fec­tive­ness, NICE al­so ad­vo­cat­ed against the use of GW Phar­ma’s $GW­PH ap­proved cannabis-de­rived spray Sativex in pa­tients with spas­tic­i­ty. In ad­di­tion, NICE said that that oth­er cannabis-based med­i­c­i­nal prod­ucts should not be of­fered to treat spas­tic­i­ty un­less as part of a clin­i­cal tri­al. NICE did, how­ev­er, en­dorse the use of syn­thet­ic cannabis treat­ment nabilone, as an add-on treat­ment for adults with chemother­a­py-in­duced nau­sea and vom­it­ing that haven’t re­spond­ed to con­ven­tion­al med­i­cine.

Paul Chrisp NICE

“We recog­nise that some peo­ple will be dis­ap­point­ed…How­ev­er, we were con­cerned when we be­gan de­vel­op­ing this guid­ance that a ro­bust ev­i­dence base for these most­ly un­li­censed prod­ucts was prob­a­bly lack­ing,” Paul Chrisp, di­rec­tor of the Cen­tre for Guide­lines at NICE, said in a state­ment. “Hav­ing now con­sid­ered all the avail­able ev­i­dence it’s there­fore not sur­pris­ing that the com­mit­tee has not been able to make many pos­i­tive rec­om­men­da­tions about their use.”

While the UK is slow­ly build­ing the ev­i­dence case to eval­u­ate the po­ten­tial med­i­c­i­nal prop­er­ties of cannabis — in the Unit­ed States, re­searchers have found that gain­ing ac­cess to cannabis for med­ical re­search has been a frus­trat­ing, up­hill bat­tle. A ma­jor­i­ty of US states have sanc­tioned the use of cannabis in a med­ical and/or recre­ation­al ca­pac­i­ty — but at the fed­er­al lev­el, it re­mains a sched­ule 1 sub­stance con­sid­ered to have no med­i­c­i­nal val­ue.

So­cial im­age: Shut­ter­stock

Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,600+ biopharma pros reading Endpoints daily — and it's free.

Novartis CEO Vas Narasimhan [via Bloomberg/Getty]

I’m not per­fect: No­var­tis chief Vas Narasimhan al­most apol­o­gizes in the wake of a new cri­sis

Vas Narasimhan has warily stepped up with what might pass as something close to a borderline apology for the latest scandal to engulf Novartis.

But he couldn’t quite get there.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,600+ biopharma pros reading Endpoints daily — and it's free.

FDA to Sarep­ta: Your wide­ly an­tic­i­pat­ed fol­lowup to Ex­ondys 51 is not get­ting an ac­cel­er­at­ed OK for Duchenne MD

In one of the least anticipated moves of the year, the FDA has rejected Sarepta’s application for an accelerated approval of its Duchenne MD drug golodirsen after fretting over safety issues.

In a statement that arrived after the bell on Monday, Sarepta explained the CRL, saying:

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,600+ biopharma pros reading Endpoints daily — and it's free.

Levi Garraway. Broad Institute via Youtube

Roche raids Eli Lil­ly for its next chief med­ical of­fi­cer as San­dra Horn­ing plans to step down

We found out Monday morning where Levi Garraway was headed after he left Eli Lilly as head of oncology R&D a few days ago. Roche named Garraway as their new chief medical officer, replacing Sandra Horning, who they say is retiring from the company.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,600+ biopharma pros reading Endpoints daily — and it's free.

Af­ter a posse of Wall Street an­a­lysts pre­dict a like­ly new win for Sarep­ta, we're down to the wire on a crit­i­cal FDA de­ci­sion

As Bloomberg notes, most of the Wall Street analysts that cover Sarepta $SRPT are an upbeat bunch, ready to cheer on the team when it comes to their Duchenne MD drugs, or offer explanations when an odd setback occurs — as happened recently with a safety signal that was ‘erroneously’ reported last week.

Ritu Baral Cowen
Endpoints News

Basic subscription required

Unlock this story instantly and join 57,600+ biopharma pros reading Endpoints daily — and it's free.

UP­DAT­ED: No­var­tis spin­off Nabri­va fi­nal­ly scores its first an­tibi­ot­ic ap­proval

In May, Nabriva Therapeutics suffered a setback after the FDA rejected its antibiotic for complicated urinary tract infections — the Novartis spinoff has now had some better luck with the US agency, which on Monday approved its other drug for community-acquired bacterial pneumonia.

The drug, lefamulin, has been developed as an intravenous and oral formulation and been tested in two late-stage clinical trials. The semi-synthetic compound, whose dosing can be switched between the two formulations, is engineered to inhibit the synthesis of bacterial protein by binding to a part of the bacterial ribosome.

Saqib Islam. CheckRare via YouTube

Spring­Works seeks $115M to push Pfiz­er drugs across fin­ish line while Sat­suma sells mi­graine play in $86M IPO

SpringWorks and Satsuma — both biotech spinouts that have closed B rounds in April — are loading up with IPO cash to boost their respective late-stage plans.
SpringWorks

Bain-backed SpringWorks is the better-known company of the two, and it’s gunning for a larger windfall of $115 million to add to $228 million from previous financings. In the process, the Stamford, CT-based team is also drawing the curtains on the partnerships it has in mind for the pair of assets it had initially licensed from Pfizer.

Mi­nor­i­ty racial groups con­tin­ue to be dis­mal­ly rep­re­sent­ed in can­cer tri­als — study

Data reveal that different racial and ethnic groups — by nature and/or nurture — can respond differently in terms of pharmacokinetics, efficacy, or safety to therapeutics, but this disparity is not necessarily accounted for in clinical trials. A fresh analysis of the last decade of US cancer drug approvals suggests the trend continues, cementing previous research that suggests oncology trials are woefully under-representative of the racial makeup of the real world.

Van­da shares slide af­ter FDA spurns their big end­point and re­jects a pitch on jet lag re­lief

Back in the spring of last year, Vanda Pharmaceuticals $VNDA served up a hot stew of mixed data for a slate of endpoints related to what they called clear evidence that their melatonin sleep drug Hetlioz (tasimelteon) could help millions of travelers suffering from jet lag.

Never mind that they couldn’t get a planned 90 people in the study, settling for 25 instead; Vanda CEO Mihael H. Polymeropoulos said they were building on a body of data to prove it would help jet-lagged patients looking for added sleep benefits. And that, they added, would be worth a major upgrade from the agency as they sought to tackle a big market.