Sanofi's sleep­ing sick­ness drug wins FDA OK and a lu­cra­tive pri­or­i­ty re­view vouch­er too

The FDA on Tues­day ap­proved the first oral treat­ment for sleep­ing sick­ness (hu­man African try­panoso­mi­a­sis) from Sanofi and the Drugs for Ne­glect­ed Dis­eases ini­tia­tive, and pro­vid­ed the part­ners with a pri­or­i­ty re­view vouch­er that they may sell for about $100 mil­lion.

Sleep­ing sick­ness is a par­a­sitic dis­ease that’s trans­mit­ted through be­ing bit­ten by tsetse flies and af­fects peo­ple liv­ing in re­mote, sub-Sa­ha­ran Africa, which puts about 65 mil­lion peo­ple at risk of in­fec­tion. But Sanofi has been able to dras­ti­cal­ly re­duce the num­ber of sleep­ing sick­ness cas­es in sub-Sa­ha­ran Africa by about 97% from 2001 and 2020, and fex­inida­zole, its oral-on­ly drug, of­fers an ad­van­tage over oth­er sleep­ing sick­ness treat­ments that re­quire in­fu­sions or in­jec­tions, and which are chal­leng­ing to ad­min­is­ter to pa­tients in re­mote parts of Africa.

“Hav­ing a sim­ple, all-oral treat­ment for sleep­ing sick­ness is a dream come true for front­line clin­i­cians,” DN­Di ex­ec­u­tive di­rec­tor Bernard Pé­coul said in a state­ment. “We are proud of this lat­est mile­stone in our long-term part­ner­ship with Sanofi, de­vel­oped in close col­lab­o­ra­tion with re­searchers in coun­tries hard-hit by sleep­ing sick­ness.”

Pa­tients take fex­inida­zole once a day for 10 days. It works to treat the first stage of the dis­ease, as well as the sec­ond, which of­ten leads to pa­tients ex­pe­ri­enc­ing neu­ropsy­chi­atric symp­toms due to par­a­sites cross­ing the blood-brain bar­ri­er.

In No­vem­ber 2018, the EMA en­dorsed fex­inida­zole as it was pushed through us­ing a non-prof­it mod­el. A group of sev­en Eu­ro­pean coun­tries joined the $62.5 mil­lion de­vel­op­ment cam­paign, and re­ceived sup­port from the Bill & Melin­da Gates Foun­da­tion and Médecins Sans Fron­tières.

Sanofi said it will pro­vide the drug to the WHO for free. And any funds earned from the PRV will be shared be­tween Sanofi and the DN­Di to fur­ther oth­er pro­grams to treat sleep­ing sick­ness and oth­er ne­glect­ed dis­eases, Sanofi said.

The trop­i­cal dis­ease PRV pro­gram, es­tab­lished in 2007, in­cen­tivizes com­pa­nies to de­vel­op new drugs for such ne­glect­ed dis­eases as tu­ber­cu­lo­sis, malar­ia, lep­rosy and oth­ers. And if a com­pa­ny wins ap­proval for a drug un­der the pro­gram, the FDA re­wards that com­pa­ny with a vouch­er that can be sold or ex­changed on the open mar­ket. Most re­cent­ly, Rhythm Phar­ma­ceu­ti­cals sold its PRV in Jan­u­ary for $100 mil­lion.

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

David Meek, new Mirati CEO (Marlene Awaad/Bloomberg via Getty Images)

Fresh off Fer­Gene's melt­down, David Meek takes over at Mi­rati with lead KRAS drug rac­ing to an ap­proval

In the insular world of biotech, a spectacular failure can sometimes stay on any executive’s record for a long time. But for David Meek, the man at the helm of FerGene’s recent implosion, two questionable exits made way for what could be an excellent rebound.

Meek, most recently FerGene’s CEO and a past head at Ipsen, has become CEO at Mirati Therapeutics, taking the reins from founding CEO Charles Baum, who will step over into the role of president and head of R&D, according to a release.

So what hap­pened with No­var­tis' gene ther­a­py group? Here's your an­swer

Over the last couple of days it’s become clear that the gene therapy division at Novartis has quietly undergone a major reorganization. We learned on Monday that Dave Lennon, who had pursued a high-profile role as president of the unit with 1500 people, had left the pharma giant to take over as CEO of a startup.

Like a lot of the majors, Novartis is an open highway for head hunters, or anyone looking to staff a startup. So that was news but not completely unexpected.

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Who are the women su­per­charg­ing bio­phar­ma R&D? Nom­i­nate them for this year's spe­cial re­port

The biotech industry has faced repeated calls to diversify its workforce — and in the last year, those calls got a lot louder. Though women account for just under half of all biotech employees around the world, they occupy very few places in C-suites, and even fewer make it to the helm.

Some companies are listening, according to a recent BIO survey which showed that this year’s companies were 2.5 times more likely to have a diversity and inclusion program compared to last year’s sample. But we still have a long way to go. Women represent just 31% of biotech executives, BIO reported. And those numbers are even more stark for women of color.

Jacob Van Naarden (Eli Lilly)

Ex­clu­sives: Eli Lil­ly out to crash the megablock­buster PD-(L)1 par­ty with 'dis­rup­tive' pric­ing; re­veals can­cer biotech buy­out

It’s taken 7 years, but Eli Lilly is promising to finally start hammering the small and affluent PD-(L)1 club with a “disruptive” pricing strategy for their checkpoint therapy allied with China’s Innovent.

Lilly in-licensed global rights to sintilimab a year ago, building on the China alliance they have with Innovent. That cost the pharma giant $200 million in cash upfront, which they plan to capitalize on now with a long-awaited plan to bust up the high-price market in lung cancer and other cancers that have created a market worth tens of billions of dollars.

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When ef­fi­ca­cy is bor­der­line: FDA needs to get more con­sis­tent on close-call drug ap­provals, agency-fund­ed re­search finds

In the exceedingly rare instances in which clinical efficacy is the only barrier to a new drug’s approval, new FDA-funded research from FDA and Stanford found that the agency does not have a consistent standard for defining “substantial evidence” when flexible criteria are used for an approval.

The research comes as the FDA is at a crossroads with its expedited-review pathways. The accelerated approval pathway is under fire as the agency recently signed off on a controversial new Alzheimer’s drug, with little precedent to explain its decision. Meanwhile, top officials like Rick Pazdur have called for a major push to simplify and clarify all of the various expedited pathways, which have grown to be must-haves for sponsors of nearly every newly approved drug.

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FDA hands ac­cel­er­at­ed nod to Seagen, Gen­mab's so­lo ADC in cer­vi­cal can­cer, but com­bo stud­ies look even more promis­ing

Biopharma’s resident antibody-drug conjugate expert Seagen has scored a clutch of oncology approvals in recent years, finding gold in what are known as “third-gen” ADCs. Now, another of their partnered conjugates is ready for prime time.

The FDA on Monday handed an accelerated approval to Seagen and Genmab’s Tivdak (tisotumab vedotin-tftv, or “TV”) in second-line patients with recurrent or metastatic cervical cancer who previously progressed after chemotherapy rather than PD-(L)1 systemic therapy, the companies said in a release.

The biggest ques­tions fac­ing gene ther­a­py, the XLMTM com­mu­ni­ty, and Astel­las af­ter fourth pa­tient death

After three patients died last year in an Astellas gene therapy trial, the company halted the study and began figuring out how to safely get the program back on track. They would, executives eventually explained, cut the dose by more than half and institute a battery of other measures to try to prevent the same thing from happening again.

Then tragically, Astellas announced this week that the first patient to receive the new regimen had died, just weeks after administration.

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Jay Bradner (Jeff Rumans for Endpoints News)

Div­ing deep­er in­to in­her­it­ed reti­nal dis­or­ders, No­var­tis gob­bles up an­oth­er bite-sized op­to­ge­net­ics biotech

Right about a year ago, a Novartis team led by Jay Bradner and Cynthia Grosskreutz at NIBR swooped in to scoop up a Cambridge, MA-based opthalmology gene therapy company called Vedere. Their focus was on a specific market niche: inherited retinal dystrophies that include a wide range of genetic retinal disorders marked by the loss of photoreceptor cells and progressive vision loss.

But that was just the first deal that whet their appetite.

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