When Cat­a­lyst Phar­ma­ceu­ti­cals’ drug for Lam­bert-Eaton myas­thenic syn­drome (LEMS) — a rare, au­toim­mune dis­or­der that af­fects the con­nec­tion be­tween nerves and mus­cles — was ap­proved last year, car­ry­ing a $375,000 an­nu­al price tag, some pa­tients were not ex­act­ly thrilled. Hun­dreds of pa­tients had been able to ac­cess a sim­i­lar drug from com­pound­ing phar­ma­cies for a frac­tion of the cost, or from a small, fam­i­ly-run New Jer­sey-based com­pa­ny called Ja­cobus Phar­ma­ceu­ti­cals for free, as part of an FDA-rat­i­fied com­pas­sion­ate use pro­gram.

Once the Cat­a­lyst treat­ment, Fir­dapse, won ap­proval for adult LEMS pa­tients, it al­so won mar­ket ex­clu­siv­i­ty span­ning sev­en years — ef­fec­tive­ly pre­clud­ing Ja­cobus and com­pound­ing phar­ma­cies from sell­ing their ver­sions.

On Mon­day, how­ev­er, the FDA may have dis­rupt­ed that sta­tus quo by ap­prov­ing the Ja­cobus ver­sion for pa­tients aged 6 to 17, based on da­ta from a 32 adult pa­tient-study. The agency said it had used adult LEMS da­ta to de­duce a safe dos­ing reg­i­men for pe­di­atric pa­tients. As far as the FDA is con­cerned, doc­tors can pre­scribe drugs for un­ap­proved use, when they judge that it is med­ical­ly ap­pro­pri­ate for their pa­tient.

Shares of Coral Gables, Flori­da-based Cat­a­lyst $CPRX cratered af­ter-mar­ket on Mon­day. On Tues­day, the stock was down 36.5% at $3.88 be­fore the bell.

“The ap­proval of Ja­cobus’ Ruzur­gi for the treat­ment of pe­di­atric pa­tients with LEMS comes as a sur­prise, as in­vestors had all but writ­ten off this ver­sion of 3,4-DAP fol­low­ing Fir­dapse’s ap­proval (for adults, who com­prise near­ly all of the LEMS pop­u­la­tion) un­der the pre­sump­tion that oth­er forms would be blocked by or­phan ex­clu­siv­i­ty. With CPRX shares -43% af­ter Mon­day’s close, the mar­ket ap­pears to be fac­tor­ing sig­nif­i­cant im­pact to Fir­dapse’s com­mer­cial prospects from off-la­bel use of a (pre­sum­ably cheap­er) agent,” Op­pen­heimer an­a­lysts wrote in a note on Tues­day.

End­points News has con­tact­ed Ja­cobus for com­ment. De­pend­ing on how Ja­cobus prices its drug, Ruzur­gi — in­sur­ers could be per­suad­ed to fa­vor it over Cat­a­lyst’s prod­uct, de­spite the fact that it is of­fi­cial­ly ap­proved for on­ly pe­di­atric use.

Ac­cord­ing to a re­port by STAT, while Ja­cobus has not so far made a de­ci­sion on pric­ing, the own­er has sug­gest­ed that the com­pa­ny had spent $60 mil­lion on R&D and man­u­fac­tur­ing, and that post-ap­proval oblig­a­tions will like­ly cost the com­pa­ny an­oth­er $10 mil­lion to $20 mil­lion.

The Ruzur­gi for­mu­la­tion re­quires re­frig­er­a­tion — while the Fir­dapse for­mu­la­tion is sta­ble at room tem­per­a­ture, which gives Cat­a­lyst a slight ad­van­tage over Ja­cobus, Sun­Trust Robin­son Humphrey an­a­lyst Ed­ward Nash wrote in a note on Tues­day. “How­ev­er, the com­mer­cial po­ten­tial for Fir­dapse now would be de­pen­dent on rel­a­tive pric­ing as well as re­im­burse­ment cov­er­age”

In LEMS pa­tients, the body’s own im­mune sys­tem launch­es an at­tack on the neu­ro­mus­cu­lar junc­tion — which con­nects nerves and mus­cles. The con­di­tion can as­so­ci­at­ed with oth­er au­toim­mune dis­eases, but tends to oc­curs in pa­tients with can­cer. It’s preva­lence in pe­di­atric pa­tients is not known, but glob­al­ly it is es­ti­mat­ed to af­fect three per mil­lion in­di­vid­u­als, ac­cord­ing to the FDA.

Fir­dapse land­ed on the US mar­ket this Jan­u­ary, and in its fourth-quar­ter earn­ings call, Cat­a­lyst said the launch was off to a strong start, with man­age­ment not­ing min­i­mal push­back from pay­ers, and in­di­cat­ing that cov­ered pa­tients pay less than $10 per month out of pock­et.

Patrick McE­nany

But the com­pa­ny’s list price had al­ready trig­gered a tem­pest of crit­i­cism in pa­tient cir­cles and in Wash­ing­ton. Ver­mont Sen­a­tor Bernie Sanders — ahead of his an­nounce­ment to make a sec­ond at­tempt at the pres­i­den­cy — spot­light­ed Cat­a­lyst for “fleec­ing” tax­pay­ers and the “im­moral ex­ploita­tion of pa­tients,” un­der­scor­ing the is­sue as yet an­oth­er in­stance of a drug com­pa­ny’s “cor­po­rate greed.”

The FDA’s de­ci­sion on Ruzur­gi was like­ly in­flu­enced by pres­sure from Sanders, Sun­Trust’s Nash not­ed.

Cat­a­lyst chief Patrick McE­nany re­butted Sander’s as­ser­tions by say­ing the biotech had spent “mil­lions” test­ing the drug; that the com­pa­ny’s pric­ing pol­i­cy is in line with ul­tra-or­phan dis­eases of sim­i­lar sever­i­ty — and the firm is do­ing its ut­most to lim­it pa­tients’ out of pock­et cost; as well as down­played Ja­cobus’ free sup­ply, say­ing it was ben­e­fit­ting not more than a few hun­dred US pa­tients.

The com­pa­ny de­clined to com­ment on the Ja­cobus ap­proval.

The top career staff at the FDA has vowed not to let politics overrule science when looking at vaccine data this fall. But Alex Azar, who happens to be their boss’s boss, apparently won’t even give them a chance to stand in the way.

In a new memorandum issued Tuesday last week, the HHS chief stripped the FDA and other health agencies under his purview of their rule making ability, asserting all such power “is reserved to the Secretary.” Sheila Kaplan of the New York Times first obtained and reported the details of the September 15 bulletin.

Having worked in parallel for years to move their respective PD-1 inhibitors up to the first-line treatment of gastrointestinal cancers, Merck and Bristol Myers Squibb finally have the data at ESMO for a showdown.

Comparing KEYNOTE-590 and CheckMate-649, of course, comes with the usual caveats. But a side-by-side look at the overall survival numbers also offer some perspective on a new frontier for the reigning checkpoint rivals, both of whom are claiming to have achieved a first.

President Donald Trump, who seems intent on announcing a COVID-19 vaccine before Election Day, could legally authorize a vaccine over the objections of experts, officials at the FDA and even vaccine manufacturers, who have pledged not to release any vaccine unless it’s proved safe and effective.

In podcasts, public forums, social media and medical journals, a growing number of prominent health leaders say they fear that Trump — who has repeatedly signaled his desire for the swift approval of a vaccine and his displeasure with perceived delays at the FDA — will take matters into his own hands, running roughshod over the usual regulatory process.

Moving into earlier and earlier treatment lines, Bristol Myers Squibb is reporting that adjuvant treatment with Opdivo has doubled the time that esophageal or gastroesophageal junction cancer patients stay free of disease.

With the CheckMate-577 data at ESMO, CMO Samit Hirawat said, the company believes it can change the treatment paradigm.

While a quarter to 30% of patients typically achieve a complete response following chemoradiation therapy and surgery, the rest do not, said Ronan Kelly of Baylor University Medical Center. The recurrence rate is also high within the first year, Hirawat added.

Does Seattle Genetics have another approval on its hands?

The last 12 months, not so great for the world, has been great for Seattle Genetics. The company landed two separate FDA approvals, signed a $4.5 billion deal with Merck and watched antibody-drug conjugates — the technology they spent years developing to broad industry skepticism — emerge suddenly as one of the most popular approaches in oncology. And on Monday at ESMO, the company and their partners at Genmab unveiled the data behind the ADC it hopes will provide its next major FDA approval.

A little under two years after a company rebranding, Immune Regulation is taking an even bigger step toward advancing its goals.

Formerly known as Peptinnovate, the British biotech announced a $53.4 million Series B early Monday morning, helping to further advance two clinical programs in rheumatoid arthritis and asthma. Though those are the two initial indications the company is focusing on, CEO Jonathan Rigby told Endpoints News he hopes the candidates can be applied to a broad swath of autoimmune disorders.

J&J’s one-two punch on EGFR-mutant non-small cell lung cancer has turned up some promising — although decidedly early — results, fueling the idea that there’s yet room to one up on third-generation tyrosine kinase inhibitors.

Twenty out of 20 advanced NSCLC patients had a response after taking a combination of an in-house TKI dubbed lazertinib and amivantamab, a bispecific antibody targeting both EGFR and cMET engineered on partner Genmab’s platform, J&J reported at ESMO. All were treatment-naïve, and none has seen their cancer progress at a median follow-up of seven months.