ICER chas­tis­es J&J for over­pric­ing de­pres­sion drug es­ke­t­a­mine ‘where there is such need for treat­ment’

Cog­nizant of the myr­i­ad of ap­proved an­ti­de­pres­sants that of­ten don’t work, reg­u­la­tors en­dorsed J&J’s $JNJ phar­ma­ceu­ti­cal ver­sion of the hal­lu­cino­genic anes­thet­ic ke­t­a­mine — es­ke­t­a­mine — in March for treat­ment-re­sis­tant de­pres­sion, well aware that the orig­i­nal cat tran­quil­iz­er is fre­quent­ly used off-la­bel for se­vere de­pres­sion. On Thurs­day, ICER con­clud­ed that while the drug, sold as Spra­va­to, does con­fer a “promis­ing” clin­i­cal ben­e­fit, its cur­rent list price ex­ceeds a com­mon cost-ef­fec­tive­ness thresh­old by a mod­est mar­gin.

In 2017, an es­ti­mat­ed 17.3 mil­lion adults in the Unit­ed States — rough­ly 7% of all US adults — had at least one ma­jor de­pres­sive episode, ac­cord­ing to the NIH. Most an­ti­de­pres­sants usu­al­ly take a few weeks to work – and half of the pa­tients fail to ful­ly re­spond. The par­ty drug (some­times re­ferred to as Kit Kat or Vi­t­a­min K) and anes­thet­ic ke­t­a­mine which can lift de­pres­sion in many pa­tients with­in hours, must be ad­min­is­tered through in­fu­sion but can have pro­found dis­so­cia­tive side-ef­fects, and pa­tients typ­i­cal­ly re­lapse af­ter treat­ment ends.

Es­ke­t­a­mine is a low-dose, nasal-spray for­mu­la­tion of ke­t­a­mine — but due to its side-ef­fect pro­file, the J&J treat­ment is de­signed to be ad­min­is­tered in the pres­ence of a health­care prac­ti­tion­er.  It was ap­proved on the ba­sis of five piv­otal Phase III stud­ies in pa­tients with treat­ment-re­sis­tant de­pres­sion.

The da­ta used to ap­prove the drug sug­gests it is clin­i­cal­ly ef­fec­tive — but with the ab­sence of long-term safe­ty da­ta, the ev­i­dence is “promis­ing but in­con­clu­sive,” ICER re­searchers said. Since there are no head-to-head tri­als com­par­ing es­ke­t­a­mine with any com­para­tors — such as ke­t­a­mine, elec­tro­con­vul­sive ther­a­py, tran­scra­nial mag­net­ic stim­u­la­tion, oral an­ti­de­pres­sants, or aug­men­ta­tion with an­tipsy­chotics (e.g., olan­za­p­ine) — its rel­a­tive ben­e­fit is al­so hard to judge, they added.

Akin to NICE in the UK, ICER is an in­de­pen­dent body that an­a­lyzes the cost-ef­fec­tive­ness of drugs and oth­er med­ical ser­vices in the Unit­ed States. Un­like NICE, though, ICER is not gov­ern­ment-af­fil­i­at­ed, but its de­ter­mi­na­tions are in­creas­ing­ly be­com­ing in­flu­en­tial with pay­ers.

ICER con­duct­ed its analy­ses us­ing two mea­sures: 1) QALYs, or qual­i­ty-ad­just­ed life-years, a mea­sure of the state of health of a per­son or group in which the ben­e­fits — in terms of length of life — are ad­just­ed to re­flect the qual­i­ty of life. Es­sen­tial­ly, one QALY is equal to one year of life in per­fect health. 2) Life years gained (LYG), which ex­press­es the ad­di­tion­al num­ber of years of life that a per­son lives as a re­sult of re­ceiv­ing treat­ment.

Com­pared with no ad­di­tion­al treat­ment be­yond a back­ground an­ti­de­pres­sant, treat­ment with es­ke­t­a­mine plus a back­ground an­ti­de­pres­sant re­sult­ed in im­por­tant QALY gains in pa­tients with treat­ment-re­sis­tant de­pres­sion (TRD), ICER said.

Us­ing the es­ke­t­a­mine list price of $295 per 28 mg in­tranasal de­vice, the treat­ment’s use re­sults in an in­cre­men­tal cost-ef­fec­tive­ness ra­tio of ap­prox­i­mate­ly $198,000 per QALY com­pared to no ad­di­tion­al treat­ment, ex­ceed­ing the com­mon­ly cit­ed cost-ef­fec­tive­ness thresh­olds of be­tween $50,000-$150,000 per QALY. Mean­while, es­ke­t­a­mine is es­ti­mat­ed to cost ap­prox­i­mate­ly $2.6 mil­lion per life year gained, ICER found.

Es­ke­t­a­mine’s ap­proval was al­so meant to en­hance ac­cess to treat­ment — since ke­t­a­mine is not cov­ered by health in­sur­ers — al­though there is a con­cern that there may still be high out-of-pock­et ex­pens­es through de­ductibles or non-cov­er­age poli­cies.

David Rind

“Es­ke­t­a­mine shows some ben­e­fits for such pa­tients and pro­vides an FDA-ap­proved treat­ment for TRD that may be cov­ered by pay­ers; how­ev­er, it is con­cern­ing to have an over­priced ther­a­py where there is such need for treat­ment. Ad­di­tion­al­ly, the sim­i­lar­i­ty of ke­t­a­mine to es­ke­t­a­mine rais­es is­sues for all stake­hold­ers about how to con­sid­er off-la­bel pre­scrip­tion and cov­er­age of a treat­ment that has not been as well stud­ied but is be­ing in­creas­ing­ly used for TRD,” said ICER’s CMO David Rind in a state­ment.

The ICER re­port was pub­lished on Thurs­day hav­ing in­cor­po­rat­ed the feed­back from pa­tient groups, clin­i­cians, drug man­u­fac­tur­ers, and oth­er stake­hold­ers to the draft ver­sion orig­i­nal­ly un­veiled in March. A fi­nal re­port is ex­pect­ed to be pub­lished in June, fol­low­ing a vote lat­er this month.

J&J dis­agrees with this re­port, a Janssen spokesper­son told End­points News. “It un­der­es­ti­mates the proven short- and long-term ben­e­fits that this treat­ment…brings to TRD pa­tients in need. The in­ac­cu­rate as­sump­tions in the draft re­port re­lat­ed to the pos­i­tive ben­e­fit risk pro­file of Spra­va­to and the com­par­i­son be­tween this FDA ap­proved treat­ment and ke­t­a­mine, a treat­ment be­ing used off-la­bel that has not been ad­e­quate­ly stud­ied and is viewed as ex­per­i­men­tal for TRD, are reck­less.”

Due to a lack of com­par­a­tive da­ta be­tween es­ke­t­a­mine and ke­t­a­mine, ICER was not able to ex­am­ine rel­a­tive cost-ef­fec­tive­ness be­tween the two ther­a­pies. In­stead, the in­sti­tute com­pared the in­di­vid­ual one-year costs and found that es­ke­t­a­mine was ten times more ex­pen­sive than ke­t­a­mine in the first year of use — de­spite the ad­min­is­tra­tion costs as­so­ci­at­ed with pro­vid­ing ke­t­a­mine in­tra­venous­ly.

2023 Spot­light on the Fu­ture of Drug De­vel­op­ment for Small and Mid-Sized Biotechs

In the context of today’s global economic environment, there is an increasing need to work smarter, faster and leaner across all facets of the life sciences industry.  This is particularly true for small and mid-sized biotech companies, many of which are facing declining valuations and competing for increasingly limited funding to propel their science forward.  It is important to recognize that within this framework, many of these smaller companies already find themselves resource-challenged to design and manage clinical studies themselves because they don’t have large teams or in-house experts in navigating the various aspects of the drug development journey. This can be particularly challenging for the most complex and difficult to treat diseases where no previous pathway exists and patients are urgently awaiting breakthroughs.

Albert Bourla, Pfizer CEO (Efren Landaos/Sipa USA/Sipa via AP Images)

Pfiz­er makes an­oth­er bil­lion-dol­lar in­vest­ment in Eu­rope and ex­pands again in Michi­gan

Pfizer is continuing its run of manufacturing site expansions with two new large investments in the US and Europe.

The New York-based pharma giant’s site in Kalamazoo, MI, has seen a lot of attention over the past year. As a major piece of the manufacturing network for Covid-19 vaccines and antivirals, Pfizer is gearing up to place more money into the site. Pfizer announced it will place $750 million into the facility, mainly to establish “modular aseptic processing” (MAP) production and create around 300 jobs at the site.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 154,000+ biopharma pros reading Endpoints daily — and it's free.

Vas Narasimhan, Novartis CEO (Thibault Camus/AP Images, Pool)

No­var­tis bol­sters Plu­vic­to's case in prostate can­cer with PhI­II re­sults

The prognosis is poor for metastatic castration-resistant prostate cancer (mCRPC) patients. Novartis wants to change that by making its recently approved Pluvicto available to patients earlier in their course of treatment.

The Swiss pharma giant unveiled Phase III results Monday suggesting that Pluvicto was able to halt disease progression in certain prostate cancer patients when administered after androgen-receptor pathway inhibitor (ARPI) therapy, but without prior taxane-based chemotherapy. The drug is currently approved for patients after they’ve received both ARPI and chemo.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 154,000+ biopharma pros reading Endpoints daily — and it's free.

Up­dat­ed: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

Yuling Li, Innoforce CEO

In­no­force opens new man­u­fac­tur­ing site in Chi­na

Innoforce is off to the races at its new site in the city of Hangzhou, China.

The Chinese CDMO announced last week that it has started manufacturing at the new facility, which was built to offer process development and manufacturing operations for RNA, plasmid DNA, viral vectors and other cell therapeutics. It will also serve as Innoforce’s corporate HQ.

The company said it’s investing more than $200 million in the 550,000-square-foot manufacturing base for advanced therapies. The GMP manufacturing facility features space for producing plasmids with three 30-liter bioreactors. For viral vector manufacturing, Innoforce also has 200- and 500-liter bioreactors at its disposal, along with eight suites to make cell therapies. The site also includes several labs and warehouse spaces.

FDA grants or­phan drug des­ig­na­tion to Al­ger­non's ifen­prodil, while ex­clu­siv­i­ty re­mains un­clear

As the FDA remains silent on orphan drug exclusivity in the wake of a controversial court case, the agency continues to hand out new designations. The latest: Algernon Pharmaceuticals’ experimental lung disease drug ifenprodil.

The Vancouver-based company announced on Monday that ifenprodil received orphan designation in idiopathic pulmonary fibrosis (IPF), a chronic lung condition that results in scarring of the lungs.  Most IPF patients suffer with a dry cough, and breathing can become difficult.

‘Catchy’ de­sign tops big ad buys on­line for grab­bing on­col­o­gists’ at­ten­tion — sur­vey

The cancer drug ads that get oncologists’ attention online are informative and use clear, eye-catching designs. That’s ZoomRx’s assessment in its most recent tracking survey, and while not necessarily surprising, the details in the research do break a few common misconceptions.

One of those is frequency, also known as the number of impressions an ad gets. No matter how many times oncologists saw a particular cancer drug ad, effectiveness prevailed in the survey across five drug brands. ZoomRx measured effectiveness as a combination of most attention-getting, relevant information and improved perception as reported by the doctors.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 154,000+ biopharma pros reading Endpoints daily — and it's free.

Tim Walbert, Horizon Therapeutics CEO (via YouTube)

And then there were two: Janssen bows out of Hori­zon takeover ne­go­ti­a­tions

Horizon Therapeutics announced last week that it was in talks with three pharmaceutical giants that could take over the company. You can now remove one of them from the equation.

J&J’s Janssen, after Horizon reported its initial involvement in early discussions to acquire the rare disease biotech, issued a statement Saturday that said Janssen “does not intend to make an offer for Horizon,” and that Janssen is bound by restrictions set in Rule 2.8 of the Irish Takeover Rules. These rules are in place for any company interested in taking over Irish companies, with Horizon Therapeutics currently based in Dublin.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 154,000+ biopharma pros reading Endpoints daily — and it's free.

Rick Modi, Affinia Therapeutics CEO

Ver­tex-part­nered gene ther­a­py biotech Affinia scraps IPO plans

Affinia Therapeutics has ditched its plans to go public in a relatively closed-door market that has not favored Nasdaq debuts for the drug development industry most of this year. A pandemic surge in 2020 and 2021 opened the doors for many preclinical startups, which caught Affinia’s attention and gave the gene therapy biotech confidence in the beginning days of 2022 to send in its S-1.

But on Friday, Affinia threw in the S-1 towel and concluded now is not the time to step onto Wall Street. The biotech has put out few public announcements since the spring of this year. Endpoints News picked the startup as one of its 11 biotechs to watch last year.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 154,000+ biopharma pros reading Endpoints daily — and it's free.