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.

It’s official. Memorial Sloan Kettering has picked a brain cancer expert as its new physician-in-chief and CMO, replacing José Baselga, who left under a cloud after being singled out by The New York Times and ProPublica for failing to properly air his lucrative industry ties.

His replacement, who now will be in charge of MSK’s cutting-edge research work as well as the cancer care delivered by hundreds of practitioners, is Lisa M. DeAngelis. DeAngelis had been chair of the neurology department and co-founder of MSK’s brain tumor center and was moved in to the acting CMO role in the wake of Baselga’s departure.

More than two years ago Ritter Pharmaceuticals managed to find enough silver lining in its Phase IIb/III study — after missing the top-line mark — to propel its lactose intolerance toward a confirmatory trial. But as it turned out, the enthusiasm only set the biotech and its investors up to be sorely disappointed.

This time around there’s little left to salvage. Not only did RP-G28 fail to beat placebo in reducing lactose intolerance symptoms, patients in the treatment group actually averaged a smaller improvement. On a composite score measuring symptoms like abdominal pain, cramping, bloating and gas, patients given the drug had a mean reduction of 3.159 while the placebo cohort saw a 3.420 drop on average (one-sided p-value = 0.0106).

An early-stage update on Adverum Biotechnologies’ intravitreal gene therapy has triggered investor concern, after patients with wet age-related macular degeneration (AMD) saw their vision deteriorate, despite signs that the treatment is improving retinal anatomy.

Adverum, on Wednesday, unveiled 24-week data from the OPTIC trial of its experimental therapy, ADVM-022, in six patients who have been administered with one dose of the therapy. On average, patients in the trial had severe disease with an average of 6.2 anti-VEGF injections in the eight months prior to screening and an average annualized injection frequency of 9.3 injections.

Too often, Alex Arfaei arrived too late. 

An analyst at BMO Capital Markets, he’d meet with biotech or pharmaceutical heads for their IPO or secondary funding and his brain, trained on a biology degree and six years at Merck and Endo, would spring with questions: Why this biomarker? Why this design? Why not this endpoint? Not that he could do anything about it. These execs were coming for clinical money; their decisions had been made and finalized long ago.

In the first of what it hopes will be a couple of major regulatory milestones for its new drug, Ardelyx has bagged an FDA approval to market Ibsrela (tenapanor) for irritable bowel syndrome.

The drug’s first application will be for IBS with constipation (IBS-C), inhibiting sodium-hydrogen exchanger NHE3 in the GI tract in such a way as to increase bowel movements and decrease abdominal pain. This comes on the heels of two successful Phase III trials.