Catch­ing Cat­a­lyst Phar­ma by sur­prise, FDA ap­proves Ja­cobus LEMS drug for pe­di­atric pa­tients — based on adult da­ta

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.

Sen. Ron Wyden (D-OR) (Francis Chung/E&E News/Politico via AP Images)

In­fla­tion re­bates in­com­ing: Wyden calls on CMS to move quick­ly as No­var­tis CEO pledges re­ver­sal

Senate Finance Chair Ron Wyden (D-OR) this week sent a letter to the head of the Centers for Medicare & Medicaid Services seeking an update on how and when new inflation-linked rebates will take effect for drugs that see major price spikes.

The newly signed Inflation Reduction Act requires manufacturers to pay a rebate to Medicare when they increase drug prices faster than the rate of inflation.

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The Big Phar­ma dis­card pile; Lay­offs all around while some biotechs bid farewell; New Roche CEO as­sem­bles top team; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

With earnings seasons in full swing, we’ve listened in on all the calls so you don’t have to. But news is popping up from all corners, so make sure you check out our other updates, too.

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Sanofi scraps PhI­II tri­al for Prin­cip­ia drug af­ter re­view­ing com­pe­ti­tion

Months after the FDA placed Phase III trials of Sanofi’s BTK inhibitor on hold, the company is winding down one of the studies.

Sanofi reported in its Q4 earnings that the URSA study “was discontinued after careful evaluation of the emerging competitive treatment landscape in” myasthenia gravis, a rare disease that causes muscle weakness.

The Phase III, placebo-controlled trial was testing tolebrutinib in patients with the moderate-to-severe form of the disease. It started in late 2021, according to records on clinicaltrials.gov, and was originally designed to recruit 154 participants who were receiving the standard of care.

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Te­va drops out of in­dus­try trade group PhRMA

Following in AbbVie’s footsteps, Teva confirmed on Friday that it’s dropping out of the industry trade group Pharmaceutical Research and Manufacturers of America (PhRMA).

Teva didn’t give a reason for its decision to leave, saying only in a statement to Endpoints News that it annually reviews “effectiveness and value of engagements, consultants and memberships to ensure our investments are properly seated.”

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Sanofi CFO Jean-Baptiste de Chatillon (L) and CEO Paul Hudson (Romuald Meigneux/Sipa via AP Images)

Sanofi sees downtick in flu sales as it preps for launch of RSV an­ti­body

Sanofi expects its RSV antibody jointly developed with AstraZeneca will be available next season, executive VP of vaccines Thomas Triomphe announced on the company’s quarterly call.

Beyfortus, also known as nirsevimab, was approved in the EU back in November and is currently under FDA review with an expected decision coming in the third quarter of this year. The news comes as the FDA plans to hold advisory committee meetings over the next couple months to review RSV vaccines from Pfizer and GSK.

Christophe Weber, Takeda CEO (Photographer: Shoko Takayasu/Bloomberg via Getty Images)

Take­da fo­cus­es on ‘di­verse’ pipeline prospects on heels of two ac­qui­si­tions

After a whopping $4 billion asset buy from Nimbus Therapeutics, along with a $400 million deal with Hutchmed for a colorectal cancer drug, Takeda executives touted pipeline optimism on its latest earnings call this week.

That’s because the TYK2 inhibitor for psoriasis Takeda is getting from Nimbus, along with the Hutchmed fruquintinib commercialization outside of China, are just two of what it reports are 10 late-stage development programs of promising candidates.

Regeneron CSO George Yancopoulos (L) and CEO Len Schleifer at a groundbreaking for its new Tarrytown, NY facility, June 2022 (Lev Radin/Pacific Press/LightRocket via Getty Images)

In show­down with Roche, Re­gen­eron gears up for po­ten­tial Eylea ex­pan­sion amid Covid de­cline

Regeneron faced a substantial slump in overall revenue last year, but the focus still remains on some of its biggest blockbusters.

The pharma with several high-profile partnerships — Sanofi and Bayer among them — said Friday that Q4 revenue was down 31% for the quarter, and down 24% for the entire year. However, that won’t stop blockbuster expansion plans.

One of those is Eylea, the Bayer-partnered eye disease drug that has been in major competition with Roche’s Vabysmo. While Eylea is currently only approved in a 2 mg dose, the company recently filed for approval to give a 8 mg dose, in hopes of making a longer-lasting treatment.

BeiGene's new website helps direct cancer patients and caregivers to a wide variety of sources for help.

BeiGene re­veals men­tal health and can­cer care gap in study, de­buts dig­i­tal re­sources

One-fourth of cancer patients are living with depression — and another 20% suffer from anxiety. That’s according to new study results from BeiGene, conducted by Cancer Support Community (CSC), about the mental and emotional health of cancer patients.

While the fact that people with cancer are also dealing with depression or anxiety may not be surprising, what is — and was to BeiGene — is that a majority of them aren’t getting support. 60% of respondents said they were not referred to a mental health professional, and even more concerning, two in five who specifically asked for mental health help did not get it. CSC, a nonprofit mental health in cancer advocacy group, surveyed more than 600 US cancer patients.

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One of the paintings from Gilead's latest campaign making AI art to help MBC patients be 'seen and heard.'

Gilead com­bines ar­ti­fi­cial in­tel­li­gence and art to draw at­ten­tion and hope to MBC

What if you could “see” the emotions and feelings of people living with metastatic breast cancer? That’s what Gilead Sciences’ agency VMLY&R Health did last year, using artificial intelligence and sound analytics to turn the interviews of three women living with metastatic triple-negative breast cancer into works of art.

Using the sound waves, a robotic painting device translated their stories of struggle and hope into colors, contours and brush strokes. The result? An art exhibition called “Paintings of Hope” that was first displayed at ESMO in September in Paris, but has since traveled to hospitals and medical conferences in Europe and Spain.

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