Pfiz­er launch­es re­bate pro­gram for rare dis­ease pa­tients who have to stop tak­ing Panzy­ga

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.

At whole­sale cost, Panzy­ga can run up a tab from around $193 to $5,790 per vial, de­pend­ing on the dose amount, ac­cord­ing to in­for­ma­tion pro­vid­ed re­cent­ly to Col­orado pre­scribers. Panzy­ga is ad­min­is­tered in two load­ing dos­es, fol­lowed by main­te­nance dos­es every three weeks.

To take the edge off, Pfiz­er says it will re­fund CIPD pa­tients for their out-of-pock­et costs for the first four treat­ments if the drug doesn’t work out, up to a max­i­mum of $16,500 per treat­ment or $50,000 to­tal.

“If your com­mer­cial in­sur­ance and/or oth­er pay­ers (“Your Plan(s)”) paid for all or a por­tion of the cost of PANZY­GA, Pfiz­er will, on your be­half, re­fund Your Plan(s) the Av­er­age Sales Price as cal­cu­lat­ed by Pfiz­er up to the Max­i­mum, less doc­u­ment­ed out-of-pock­et pay­ments pro­vid­ed by you or on your be­half,” the com­pa­ny’s web­site states.

While Pfiz­er’s re­bate pro­gram for its can­cer drug Xalko­ri is avail­able to Medicare pa­tients, the Panzy­ga pro­gram is on­ly avail­able to com­mer­cial­ly-in­sured pa­tients or those who pay cash, ex­clud­ing pa­tients cov­ered by gov­ern­ment pro­grams such as Medicare and Med­ic­aid.

Un­der the Xalko­ri pro­gram, el­i­gi­ble pa­tients can get their out-of-pock­et costs cov­ered if they dis­con­tin­ue the drug be­fore the fourth 30-day sup­ply is ad­min­is­tered.

The Panzy­ga re­bate on­ly ap­plies if the drug was dis­con­tin­ued for “clin­i­cal rea­sons de­fined at the dis­cre­tion of the health­care provider,” ac­cord­ing to Pfiz­er. Pa­tients who stop tak­ing the drug by choice or be­cause of af­ford­abil­i­ty don’t qual­i­fy. Pa­tients are al­so dis­qual­i­fied if they’ve used an­oth­er im­munoglob­u­lin prod­uct in the past 12 months.

“Ac­cess to treat­ment for Chron­ic In­flam­ma­to­ry De­myeli­nat­ing Polyneu­ropa­thy may be a bar­ri­er for pa­tients across the U.S.,” a Pfiz­er spokesper­son said in an emailed state­ment. “As part of our com­mit­ment to pa­tients and be­lief in our med­i­cines, Pfiz­er is proud to of­fer the Pfiz­er Pledge War­ran­ty Pro­gram for PANZY­GA. PANZY­GA, ap­proved by the U.S. FDA for the treat­ment of CIDP, has an es­tab­lished clin­i­cal pro­file and HCPs are able to as­sess pa­tient re­sponse with­in the first few treat­ments ad­min­is­tered.”

The news comes as the Sen­ate re­cent­ly passed ma­jor drug pric­ing re­form, which would en­able Medicare ne­go­ti­a­tions and cap se­niors’ out-of-pock­et costs at $2,000 per year. While Pres­i­dent Joe Biden tout­ed the leg­is­la­tion as a “crit­i­cal step” that would “help Amer­i­cans save mon­ey on pre­scrip­tion drugs, health pre­mi­ums, and much more,” phar­ma ex­ecs have ar­gued that the bill will stymie in­no­va­tion.

The Fac­tors Dri­ving a Rapid Evo­lu­tion of Gene & Cell Ther­a­py and CAR-T Clin­i­cal Re­search in APAC

APAC is the fastest growing region globally for cell & gene therapy trials representing more than a third of all cell & gene studies globally, with China leading in the region. 

APAC is the leading location globally for CAR-T trials with China attracting ~60% of all CAR-T trials globally between 2015-2022. The number of CAR-T trials initiated by Western companies has rapidly increased in recent years (current CAGR of about 60%), with multiple targets being explored including CD19, CD20, CD22, BCMA, CD30, CD123, CD33, CD38, and CD138.

The End­points 11; blue­bird's $3M gene ther­a­py; Bio­gen tout new neu­ro da­ta; Harsh re­views for can­cer drugs; 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.

Reading about John Carroll’s pick of biotech’s most promising startups has become a treasured tradition. If you ever get curious about previous classes of the Endpoints 11, you can find all of them (plus a number of our other regular specials) here.

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EMA warns of short­ages of two Boehringer heart drugs due to a spike in de­mand

The EMA is putting EU member states on alert over the shortage of two drugs that counter heart attacks due to an uptick in demand.

On Friday, the EMA sent out a warning that two Boehringer Ingelheim drugs are experiencing a shortage: Actilyse and Metalyse. The drugs are used as emergency treatments for adults experiencing acute myocardial infarction, or a heart attack, by dissolving blood clots that have formed in the blood vessels.

The End­points 11: The top pri­vate biotechs in pur­suit of new drugs. Push­ing the en­ve­lope with pow­er­ful new tech­nolo­gies

Right around the beginning of the year, we got a close-up look at what happens after a boom ripples through biotech. The crash of life sciences stocks in Q1 was heard around the world.

In the months since, we’ve seen the natural Darwinian down cycle take effect. Reverse mergers made a comeback, with more burned out shells to go public at a time IPOs and road shows are out of favor. And no doubt some of the more recent arrivals on the investing side of the business are finding greener pastures.

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Solicitor General Elizabeth Prelogar

Should SCO­TUS hear Am­gen's Repatha case? So­lic­i­tor gen­er­al says no

Back in April, Amgen said it was encouraged by the solicitor general’s anticipated review of its Supreme Court petition to rehear a Repatha patent case. They’re likely much less optimistic about the outcome now.

Solicitor General Elizabeth Prelogar wrote in a recent 27-page brief that Amgen’s arguments “lack merit and further review is not warranted.”

The case traces back to a suit filed in 2014 against Sanofi and Regeneron’s Praluent, which ended up beating Amgen’s PCSK9 blockbuster Repatha to market by a month just a year later.

Klick Health gath­ers biotech and phar­ma lu­mi­nar­ies to dis­cuss in­dus­try in­no­va­tions, in­vest­ments and fu­ture

At Klick Health’s first Ideas Exchange conference with biotech and pharma industry insiders since before the pandemic began, it was no surprise many conversations included Covid topics. Yet while vaccines and treatments were discussed, so too were the effects on drug development, federal responses, health inequities — and what to do now and next.

George Yancopoulos, chief scientist and cofounder of Regeneron, opened the conference responding to a question from Acorda CEO Ron Cohen about the spotlight on the industry during Covid and some of the “flak” biopharma has taken in the past.

FDA's out­side ex­perts vote in fa­vor of Fer­ring's fe­cal trans­plant for C. dif­fi­cile, set­ting the stage for Seres

FDA’s outside advisors voted in favor of Ferring Pharmaceuticals’ RBX2660, an experimental poop-based drug implant that the company says would be the first microbiota-based live biotherapeutic to receive an FDA green light.

That was a point repeatedly discussed during the Vaccines and Related Biological Products Advisory Committee, or VRBPAC, meeting Thursday when evaluating Ferring’s fecal microbiota transplant, or FMT, for reducing the recurrence of Clostridioides difficile infection in adults who have received antibiotics. Multiple members brought up the need for a regulated product amid a landscape of unregulated FMTs already happening in clinical care.

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Vas Narasimhan, Novartis CEO (Simon Dawson/Bloomberg via Getty Images)

No­var­tis un­veils 'US-first' strat­e­gy ahead of San­doz spin­off

Weeks after announcing the spinoff of generics arm Sandoz, Vas Narasimhan paints a picture of the new, slimmer Novartis — with a “US-first mindset,” he said at an investor event on Thursday.

The CEO unveiled ambitious plans to become a top-five player in the US by 2027 at Novartis’ “Meet the Management” event in Basel, Switzerland, which means ramping up clinical trials in the states and “building capability and talent, among other things.” The company’s also shooting for a top-three ranking in China.

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Pfiz­er sacks phar­ma com­pe­ti­tion in ear­ly NFL TV ad­ver­tis­ing sea­son

If pharma advertising had a fantasy football league, Pfizer would be crushing the competition. A dive into the National Football League’s TV commercial buys across early season games by iSpot shows a hefty lead with its Covid-19 Comirnaty vaccine ads.

More than 175 million impressions with $9.5 million in media spending put Pfizer in the top spot with a 65% share of voice across NFL pharma spending, according to the real-time TV ad tracker. In a distant second place is Bristol Myers Squibb’s Opdivo with 44 million impressions, $5.2 million in spending and a 16% share, followed by BMS’ Zeposia with 31 million impressions, $3.3 million in media buys and an 11% share.

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