Stick­er shock: No­var­tis says its top drug prospect is worth $4M-$5M for a once-and-done use

Val­ue and pric­ing in life-long rare dis­ease treat­ment. (No­var­tis, page 48)

AveX­is man­aged to grab the in­dus­try’s at­ten­tion when re­searchers post­ed ear­ly and stun­ning re­sults for their life-sav­ing gene ther­a­py to treat rare cas­es of spinal mus­cu­lar at­ro­phy. They were back in the spot­light when No­var­tis reached out to buy the com­pa­ny for $8.7 bil­lion. And now they’re mak­ing a re­turn trip to cen­ter stage with an ar­gu­ment that their once-and-done ap­proach to SMA — AVXS-101 — could jus­ti­fi­ably be priced at $4 mil­lion to $5 mil­lion.

Dave Lennon (LinkedIn)

“Four mil­lion dol­lars is a sig­nif­i­cant amount of mon­ey, but we be­lieve this is a cost-ef­fec­tive point,” Dave Lennon, pres­i­dent of AveX­is told in­vestors ear­li­er to­day, ac­cord­ing to a re­port from Reuters. “We’ve shown through oth­er stud­ies we are cost ef­fec­tive in the range of $4-$5 mil­lion. And ul­ti­mate­ly, this is im­por­tant con­text as we con­sid­er how we’re go­ing to eval­u­ate val­ue for (the ther­a­py called) AVXS-101.”

No­var­tis out­lined their ar­gu­ment in a slide that used price guide­lines from the cost watch­dogs at ICER and NICE to make their case. ICER, for ex­am­ple, backed Roche’s $482,000 first-year price for Hem­li­bra, with a 10-year cost of $4.5 mil­lion. But it’s hard to see ei­ther group go­ing for $4 mil­lion worth of stick­er shock for a one-time ther­a­py. ICER re­ject­ed Spark’s $850,000 price tag for its gene ther­a­py for a rare form of blind­ness as ex­treme and need­ed to be cut by at least 50% to re­flect its true val­ue to pa­tients. And NICE has had no prob­lem re­ject­ing oth­er rare dis­ease drugs with much small­er prices at­tached.

No­var­tis may not ac­tu­al­ly be plan­ning to try and col­lect $4 mil­lion for their drug, and ex­ecs de­murred on pro­ject­ing the cost to­day. But they seem in­tent on soft­en­ing up the pay­er mar­ket for some­thing sig­nif­i­cant­ly high­er than $1 mil­lion — once con­sid­ered a jaw-drop­ping, break-the-bank sum.

And that would put them at the very top of the heap of the most ex­pen­sive drugs on the plan­et.

In their fa­vor: Ri­vals at Bio­gen charge $750,000 for the first year of Spin­raza, then $375,000 a year af­ter that — for life. That’s $1.87 mil­lion over 4 years. And they don’t get much grief for it.

At the very least, you can ex­pect a whole new dis­cus­sion around the pric­ing of gene ther­a­pies, as de­vel­op­ers dis­cuss dif­fer­ent pay plans to spread out the cost or of­fer var­i­ous mon­ey-back guar­an­tees.

Biogen CEO Michel Vounatsos (via Getty Images)

With ad­u­canum­ab caught on a cliff, Bio­gen’s Michel Vounatsos bets bil­lions on an­oth­er high-risk neu­ro play

With its FDA pitch on the Alzheimer’s drug aducanumab hanging perilously close to disaster, Biogen is rolling the dice on a $3.1 billion deal that brings in commercial rights to one of the other spotlight neuro drugs in late-stage development — after it already failed its first Phase III.

The big biotech has turned to Sage Therapeutics for its latest deal, close to a year after the crushing failure of Sage-217, now dubbed zuranolone, in the MOUNTAIN study.

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As­traZeneca, Ox­ford on the de­fen­sive as skep­tics dis­miss 70% av­er­age ef­fi­ca­cy for Covid-19 vac­cine

On the third straight Monday that the world wakes up to positive vaccine news, AstraZeneca and Oxford are declaring a new Phase III milestone in the fight against the pandemic. Not everyone is convinced they will play a big part, though.

With an average efficacy of 70%, the headline number struck analysts as less impressive than the 95% and 94.5% protection that Pfizer/BioNTech and Moderna have boasted in the past two weeks, respectively. But the British partners say they have several other bright spots going for their candidate. One of the two dosing regimens tested in Phase III showed a better profile, bringing efficacy up to 90%; the adenovirus vector-based vaccine requires minimal refrigeration, which may mean easier distribution; and AstraZeneca has pledged to sell it at a fraction of the price that the other two vaccine developers are charging.

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John Maraganore, Alnylam CEO (Scott Eisen/Bloomberg via Getty Images)

Al­ny­lam gets the green light from the FDA for drug #3 — and CEO John Maraganore is ready to roll

Score another early win at the FDA for Alnylam.

The FDA put out word today that the agency has approved its third drug, lumasiran, for primary hyperoxaluria type 1, better known as PH1. The news comes just 4 days after the European Commission took the lead in offering a green light.

An ultra rare genetic condition, Alnylam CEO John Maraganore says there are only some 1,000 to 1,700 patients in the US and Europe at any particular point. The patients, mostly kids, suffer from an overproduction of oxalate in the liver that spurs the development of kidney stones, right through to end stage kidney disease.

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Jason Kelly, Ginkgo Bioworks CEO (Kyle Grillot/Bloomberg via Getty Images)

Af­ter Ko­dak de­ba­cle, US lends $1.1B to a syn­thet­ic bi­ol­o­gy com­pa­ny and their big Covid-19, mR­NA plans

In mid-August, as Kodak’s $765 million government-backed push into drug manufacturing slowly fell apart in national headlines, Ginkgo Bioworks CEO Jason Kelly got a message from his company’s government liaison: HHS wanted to know if they, too, might want a loan.

The government’s decision to lend Kodak three quarters of a billion dollars raised eyebrows because Kodak had never made drugs before. But Ginkgo, while not a manufacturing company, had spent the last decade refining new ways to produce materials inside cells and building automated facilities across Boston.

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Bax­ter con­tin­ues on-shoring push with $50M In­di­ana ex­pan­sion

It’s been a banner year for the once humdrum business of manufacturing drugs, particularly vaccines. Billions have been spent ramping up facilities for Covid-19 jabs, while individual CDMOs have expanded their facilities, apparently anticipating demand or responding to a government-led push to onshore drug manufacturing.

Now Baxter Biopharma Solutions, the CDMO wing of the many-armed healthcare giant Baxter, is getting in on the game. On Tuesday, they announced plans to spend $50 million to expand their flagship, 600,000 square-foot facility in Bloomington, IN.

Eu­ro­pean Union aims to es­tab­lish patent workaround in case of emer­gen­cies while try­ing to strength­en its own IP

The European Union is looking at ways to bypass patent protections and make it easier to make generic drugs in cases of emergency such as the Covid-19 pandemic, a new document says.

Normally, under WTO regulations, the practice known as “compulsory licensing” is allowed in exceptional circumstances and could be applied as a waiver to bypass patent holders. Wednesday’s document was published as part of the EU’s plan to shore up the intellectual property rights of its member states.

Pur­due Phar­ma pleads guilty in fed­er­al Oxy­Con­tin probe, for­mal­ly rec­og­niz­ing it played a part in the opi­oid cri­sis

Purdue Pharma, the producer of the prescription painkiller OxyContin, admitted Tuesday that, yes, it did contribute to America’s opioid epidemic.

The drugmaker formally pleaded guilty to three criminal charges, the AP reported, including getting in the way of the DEA’s efforts to combat the crisis, failing to prevent the painkillers from ending up on the black market and encouraging doctors to write more painkiller prescriptions through two methods: paying them in a speakers program and directing a medical records company to send them certain patient information. Purdue’s plea deal calls for $8.3 billion in criminal fines and penalties, but the company is only liable for a fraction of that total — $225 million.

PhRMA sues Trump gov­ern­ment over drug im­por­ta­tion rule — days be­fore it's set to be ef­fec­tive

Ever since President Donald Trump floated the idea of using state-sponsored importation to lower drug prices, PhRMA has made its opposition abundant. Not only is the proposal dangerous and futile,  but the trade group has also argued that it may even be illegal.

Now that the FDA has issued its final rule permitting states to bring certain drugs from Canada, PhRMA is taking the government to court — just a few days before the rule is slated to take effect.

The ad­u­canum­ab co­nun­drum: The PhI­II failed a clear reg­u­la­to­ry stan­dard, but no one is cer­tain what that means any­more at the FDA

Eighteen days ago, virtually all of the outside experts on an FDA adcomm got together to mug the agency’s Billy Dunn and the Biogen team when they presented their upbeat assessment on aducanumab. But here we are, more than 2 weeks later, and the ongoing debate over that Alzheimer’s drug’s fate continues unabated.

Instead of simply ruling out any chance of an approval, the logical conclusion based on what we heard during that session, a series of questionable approvals that preceded the controversy over the agency’s recent EUA decisions has come back to haunt the FDA, where the power of precedent is leaving an opening some experts believe can still be exploited by the big biotech.

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