Faster, prici­er and of­ten worth­less: Study slams reg­u­la­tors for wave of ques­tion­able can­cer drug OKs

The ex­plo­sion of new can­cer drugs that’s oc­curred over the past decade in­cludes some ma­jor stand­outs that in­clude ther­a­pies that have reg­is­tered some jaw-drop­ping re­sults in clin­i­cal tri­als. But when re­searchers at King’s Col­lege Lon­don and the Lon­don School of Eco­nom­ics took a step back and ex­am­ined 68 can­cer in­di­ca­tions ap­proved in Eu­rope over a 5-year pe­ri­od through 2013, they found that many were OK’d on on­ly flim­sy da­ta de­rived from un­re­li­able tri­al de­signs and at least 10 of these OKs have nev­er proved to have any re­al val­ue for pa­tients.

Kick­ing back at in­dus­try’s crit­i­cism of groups like NICE, which of­ten balk at cov­er­ing high-priced can­cer drugs, the bio­phar­ma crit­ics say that the vogue of fast-track­ing on­col­o­gy ther­a­pies has put a long line­up of drugs in­to reg­u­lar use, even though the man­u­fac­tur­ers haven’t pro­duced sol­id ev­i­dence of ef­fi­ca­cy four or more years lat­er.

Their study was pub­lished in the BMJ.

Court­ney Davis

The two au­thors — Court­ney Davis at King’s Col­lege in Lon­don, and Huseyin Naci of LSE Health — say 39 in­di­ca­tions (57%) were sanc­tioned “with­out ev­i­dence of a sur­vival or qual­i­ty of life ben­e­fit.” And they ex­co­ri­at­ed the use of sur­ro­gate end­points, say­ing they are un­re­li­able at best.

While 24 of these 68 in­di­ca­tions of­fered ev­i­dence for in­creased sur­vival, the av­er­age was less than 3 months. And on­ly 7 of 68 pro­vid­ed da­ta demon­strat­ing that they im­proved qual­i­ty of life.

“When ex­pen­sive drugs that lack clin­i­cal­ly mean­ing­ful ben­e­fits are ap­proved and re­im­bursed with­in pub­licly fund­ed health­care sys­tems, in­di­vid­ual pa­tients may be harmed, im­por­tant re­sources wast­ed, and the de­liv­ery of eq­ui­table and af­ford­able care is un­der­mined,” the re­searchers say. They added that while reg­u­la­tors have the au­thor­i­ty to pull a drug back from the mar­ket if it doesn’t demon­strate ef­fi­ca­cy, they don’t do it.

Can­cer Re­search UK cau­tioned, though, that the sys­tem on ap­prov­ing and pay­ing for can­cer drugs has un­der­gone some change in re­cent years, with a greater em­pha­sis on some of the weak spots high­light­ed in the study.

Of course, many of these same drugs are al­so ap­proved in the US. And the au­thors’ list of ques­tion­able drugs cov­ers some block­busters. It in­cludes:

→ Avastin, OK’d for ovar­i­an can­cer by the EU in 2011, they say, slap­ping re­searchers for switch­ing from over­all sur­vival to pro­gres­sion-free sur­vival.

Vinay Prasad Wash­ing­ton Post

→ Her­ceptin al­so comes in for crit­i­cism, with the re­searchers flag­ging an ap­proval for breast can­cer long be­fore it failed to demon­strate a sur­vival ben­e­fit.

→ Cel­gene’s Po­m­a­lyst was OK’d in Eu­rope af­ter reg­u­la­tors crit­i­cized their com­par­i­son of the drug com­bined with low dose dex­am­etha­sone against high dose dex­am­etha­sone.

“The ex­pense and tox­i­c­i­ty of can­cer drugs means we have an oblig­a­tion to ex­pose pa­tients to treat­ment on­ly when they can rea­son­ably ex­pect an im­prove­ment in sur­vival or qual­i­ty of life,” in­dus­try crit­ic Vinay Prasad told CRUK. The find­ings sug­gest “we may be falling far short of this im­por­tant bench­mark”

Forge Bi­o­log­ics’ cGMP Com­pli­ant and Com­mer­cial­ly Vi­able Be­spoke Affin­i­ty Chro­matog­ra­phy Plat­form

Forge Biologics has developed a bespoke affinity chromatography platform approach that factors in unique vector combinations to streamline development timelines and assist our clients in efficiently entering the clinic. By leveraging our experience with natural and novel serotypes and transgene conformations, we are able to accelerate affinity chromatography development by nearly 3-fold. Many downstream purification models are serotype-dependent, demanding unique and time-consuming development strategies for each AAV gene therapy product1. With the increasing demand to propel AAV gene therapies to market, platform purification methods that support commercial-scale manufacturing of high-quality vectors with excellent safety and efficacy profiles are essential.

Cy­to­ki­net­ics’ ALS drug fails PhI­II, leav­ing the biotech with a sin­gle late-stage prospect

Cytokinetics’ candidate for the muscle disease amyotrophic lateral sclerosis, or ALS, failed a Phase III trial, the Bay Area biotech announced Friday morning.

At a second interim analysis of the trial, an independent review committee recommended that Cytokinetics discontinue its COURAGE-ALS trial for reldesemtiv, as it “found no evidence of effect” compared to placebo on the primary or key secondary endpoints.

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CHMP gives thumbs-up for We­govy use in ado­les­cents, along with nine new drug rec­om­men­da­tions

The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) recommended nine drugs for approval this week while also giving thumbs up for six expanded indications, including Novo Nordisk’s approved obesity medication Wegovy for younger people. Wegovy is already approved as an obesity treatment in the EU for adults, and the new indication would allow prescriptions for adolescents aged 12 and older.

Green­Light re­ceives buy­out of­fer; Apol­lomics com­pletes SPAC merg­er

RNA biotech GreenLight Biosciences has been handed an offer for potential acquisition.

GreenLight said in a release that it has received a non-binding “indication of interest” from Fall Line Endurance Fund to acquire GreenLight’s capital stock for $0.60 per share in cash. The release said any potential agreement between the two parties would depend on certain conditions.

Through a special committee, the biotech will evaluate the offer but added there’s no certainty a deal will go forward. GreenLight will also not make any more announcements until a deal comes through or “otherwise determines” a statement is necessary.

Mathai Mammen, FogPharma's next CEO

Math­ai Mam­men hands in J&J's R&D keys to lead Greg Ver­dine’s Fog­Phar­ma 

In the early 1990s, Mathai Mammen was a teaching assistant in Greg Verdine’s Science B46 course at Harvard. In June, the former R&D head at Johnson & Johnson will succeed Verdine as CEO, president and chair of FogPharma, the same month the seven-year-old biotech kickstarts its first clinical trial.

After leading R&D at one of the largest drugmakers in the world, taking the company through more than half a dozen drug approvals in the past few years, not to mention a Covid-19 vaccine race, Mammen departed J&J last month and will take the helm of a Cambridge, MA biotech attempting to go after what Verdine calls the “true emperor of all oncogenes” — beta-catenin.

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Sen­ate Fi­nance Com­mit­tee lobs more bi­par­ti­san pres­sure on­to PBMs

Congress is honing in on how it wants to overhaul the rules of the road for pharmacy benefit managers, with a Senate Finance Committee hearing Thursday serving as the latest example of the Hill’s readiness to make changes to how pharma middlemen operate.

While pledging to ensure patients and pharmacies “don’t get a raw deal,” Finance Committee Chair Ron Wyden (D-OR) laid out the beginning of what looks like a major bipartisan effort — moves the PBM industry is likely to challenge vigorously.

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Nicklas Westerholm, Egetis Therapeutics CEO

Ac­qui­si­tion talks on­go­ing for Swedish rare dis­ease biotech Egetis, shares up al­most 40%

Shares of the Sweden-based rare disease biotech Egetis Therapeutics skyrocketed on Thursday afternoon as the company said it’s engaged in “ongoing discussion” with external parties regarding a “potential acquisition.”

Egetis confirmed rumors with a statement on Thursday while noting that there is no certainty that a takeover offer will be made.

Nonetheless, the possibility of an acquisition has shot up Egetis’ share price. By the afternoon on Thursday, its stock price was {$EGTX.ST} up over 38%. An Egetis spokesperson told Endpoints News in an email that it has no further comments.

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Lu­pus drug de­vel­op­ment mar­ket heat­ing up, while FDA links with ad­vo­ca­cy group to fur­ther ac­cel­er­ate re­search

The long-underserved systemic lupus erythematosus (SLE) market is suddenly buzzing with treatment possibilities. Less than two years after AstraZeneca’s approval for Saphnelo — the first new SLE drug in a decade and joining just one other approved in GSK’s Benlysta – the pipeline of potential drugs numbers in the dozens.

Although most are very early stage — Spherix Global Insights estimates five in Phase II/III — the pharma R&D enthusiasm is catching on among doctors, patients and advocacy groups. On Wednesday, the Lupus Research Alliance and the FDA formed a novel private-public partnership called Lupus Accelerating Breakthroughs Consortium (Lupus ABC) to help advance lupus clinical trial success.

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Stéphane Bancel, Moderna CEO (AP Photo/Markus Schreiber)

Mod­er­na so­lid­i­fies deal with Kenya to build mR­NA man­u­fac­tur­ing fa­cil­i­ty

The mRNA player Moderna is further cementing its presence on the African continent.

Moderna announced on Thursday that it has finalized an agreement with Kenya’s government to partner up and bring an mRNA manufacturing facility to the east African nation. The new facility aims to manufacture up to 500 million doses of vaccines annually. Moderna also said the new facility will have the ability to spike its production capabilities to respond to public health emergencies on the continent or globally.