Some­body high up at the FDA must re­al­ly like Karyopharm $KP­TI.

On Wednes­day the biotech an­nounced that the agency had stamped an OK on their ap­pli­ca­tion to mar­ket se­linex­or for mul­ti­ple myelo­ma, cut­ting ahead of the late-stage re­sults — though in an in­trigu­ing note the agency said they had a look at ad­di­tion­al re­sults from an on­go­ing study.

The ap­proval came de­spite a sol­id ma­jor­i­ty of ex­perts on an out­side pan­el who vot­ed against a quick OK, pre­fer­ring to see the Phase III da­ta first. And it ar­rives de­spite an in­ter­nal re­view com­plete with a host of ob­jec­tions to the da­ta de­liv­ered to back the pitch.

The drug will be sold as Xpovio at a whole­sale price of $22,000 a month.

Pre­sent­ed with the sur­prise turn­around, in­vestors bid up the shares by 36% as a copy of the la­bel spread on Twit­ter ahead of the re­lease.

FDA rep­re­sen­ta­tives were po­lite about it, but out­lined mul­ti­ple prob­lems with the da­ta that Karyopharm pre­sent­ed: Miss­ing da­ta due to dropouts, un­ac­cept­able re­al world ev­i­dence, an ab­sence of ev­i­dence of pos­i­tive sin­gle agent ac­tiv­i­ty (there are neg­a­tive re­sults), most­ly par­tial re­spons­es and much, much more — all in­for­ma­tion that the biotech failed to spot­light in the lead-up to the NDA. In one study the FDA cit­ed, the over­all sur­vival rate was worse in the se­linex­or arm.

David Har­ring­ton [Dana Far­ber]

Then there was the tox­i­c­i­ty pro­file, with a high fre­quen­cy of treat­ment emer­gent ad­verse events among pa­tients tak­ing the drug. The agency cit­ed a 94% rate of grade 3 or grade 4 ad­verse event. 10 deaths were due to a fa­tal ad­verse event in the main sin­gle arm study used for the ac­cel­er­at­ed ap­proval. And 9 in 10 pa­tients re­quired a dose mod­i­fi­ca­tion, with a ma­jor­i­ty re­quir­ing 2 mod­i­fi­ca­tions.

That case per­suad­ed 8 of 13 ex­perts to vote against an ear­ly ap­proval. But the 5 votes in fa­vor il­lus­trat­ed the agency’s ap­petite for new drugs for pa­tients who have run out of op­tions.

Dana Far­ber’s David Har­ring­ton joined the mi­nor­i­ty in fa­vor of pro­vid­ing an ac­cel­er­at­ed ap­proval. “The da­ta are not con­clu­sive in ei­ther di­rec­tion,” he said at the time, but…”I think we do pa­tients some po­ten­tial ben­e­fit if this is used con­struc­tive­ly.”

They’ll have that now. The drug is re­served for the last line of de­fense af­ter at least 4 pri­or drugs.

Over the past three years, Endpoints News has spotlighted 60 women who have blazed trails and supercharged R&D across the biopharma world. And judging from the response we’ve received, to both our special reports and live events, telling their stories — including any obstacles they may have had to overcome — has inspired our readers in many different ways.

But change takes time, and the fact remains that women are still underrepresented at the upper ranks of the drug-making world.

The FDA is still wary about a trial testing a microbiome therapy in patients with steroid-resistant acute graft-versus-host disease (aGVHD).

A year after MaaT Pharma’s IND application in the US was first met with a clinical hold, the French biotech said the agency is maintaining the hold. The crux of the matter, MaaT suggested, has to do with the way it puts together its drug candidate, which is administered as an enema (i.e. an injection of fluid into the bowel).

Pfizer is launching its second-ever rebate program, this time for Panzyga, its treatment for a rare neurological disease of the peripheral nerves.

The program began last month, according to STAT which first reported the news, and offers a refund of out-of-pocket costs for patients who must discontinue their course before the fifth treatment for “clinical reasons.”

Panzyga was approved back in 2018 to treat primary immunodeficiency (PI) in patients two years and older and chronic immune thrombocytopenia (cITP) in adults. It has since picked up an indication in chronic inflammatory demyelinating polyneuropathy (CIDP), a condition that’s characterized by weakness of the arms or legs, tingling or numbness, and a loss of deep tendon reflexes, according to the NIH.