FDA hits a green light on 2 more rare dis­ease drugs, giv­ing Alex­ion an ear­ly shot at pro­tect­ing PNH fran­chise

Faced with the prospect of a par­tial gov­ern­ment shut­down lat­er to­day, the FDA has pulled the trig­ger on a pair of new drug ap­provals.

Alex­ion got its nod 2 months ear­ly for ALXN1210 for parox­ys­mal noc­tur­nal he­mo­glo­bin­uria, or PNH, which will now be sold as Ul­tomiris. And Stem­line Ther­a­peu­tics won an FDA ap­proval to mar­ket El­zon­ris (tagrax­o­fusp-erzs) for rare cas­es of blas­tic plas­ma­cy­toid den­drit­ic cell neo­plasm, or BPD­CN.

The FDA has now ap­proved 59 new drugs through CDER in 2018, part of its record-set­ting year as the in­dus­try en­joys a surge of new mar­ket­ing OKs.

Ge­of­frey Porges

Alex­ion $ALXN is plan­ning to price Ul­tomiris at a dis­count to Soliris, long one of the most ex­pen­sive ther­a­pies in the world. ALXN1210 proved just as ef­fec­tive as their big drug Soliris in treat­ing PNH, with less fre­quent dos­ing that will now pave the way to a dis­count price. Leerink’s Ge­of­frey Porges counts him­self as a sup­port­er of this strat­e­gy ahead of prospec­tive com­peti­tors for their PNH fran­chise.

While in­vestors may re­act neg­a­tive­ly to the ini­tial im­pact of this pric­ing head­wind (rev­enue for the PNH in­di­ca­tion for es­tab­lished pa­tients down 10%), we be­lieve the stock al­ready dis­counts sub­stan­tial price ero­sion and mar­ket share loss to com­peti­tors. In­stead, Alex­ion’s de­ci­sion to move to­ward more sus­tain­able pric­ing with a su­pe­ri­or prod­uct should front-run and counter these po­ten­tial risks, and build to­ward a more sta­ble rev­enue tra­jec­to­ry and longevi­ty than cur­rent­ly ex­pect­ed. 

Stem­line $STML, mean­while, had to en­dure a ker­fuf­fle ear­li­er on re­gard­ing its tardy ac­knowl­edge­ment of a pa­tient death dur­ing a small study of their drug, but the agency nonethe­less pro­vid­ing a thumbs-up on a small set of da­ta.

The drug was waved through af­ter post­ing wide­ly vary­ing re­sults from two pa­tient co­horts which re­ceived the drug in a tiny sin­gle-arm study.

The ef­fi­ca­cy of El­zon­ris was stud­ied in two co­horts of pa­tients in a sin­gle-arm clin­i­cal tri­al. The first tri­al co­hort en­rolled 13 pa­tients with un­treat­ed BPD­CN, and sev­en pa­tients (54%) achieved com­plete re­mis­sion (CR) or CR with a skin ab­nor­mal­i­ty not in­dica­tive of ac­tive dis­ease (CRc). The sec­ond co­hort in­clud­ed 15 pa­tients with re­lapsed or re­frac­to­ry BPD­CN. One pa­tient achieved CR and one pa­tient achieved CRc.

The OK comes with a black box warn­ing alert­ing physi­cians to be on alert for a host of po­ten­tial side ef­fects, but this is one con­di­tion where there are no ap­proved drugs, putting it on a pri­or­i­ty list for the FDA, which of­fered its break­through drug des­ig­na­tion to help speed it along.

Grow­ing ac­cep­tance of ac­cel­er­at­ed path­ways for nov­el treat­ments: but does reg­u­la­to­ry ap­proval lead to com­mer­cial suc­cess?

By Mwango Kashoki, MD, MPH, Vice President-Technical, and Richard Macaulay, Senior Director, of Parexel Regulatory & Access

In recent years, we’ve seen a significant uptake in the use of regulatory options by companies looking to accelerate the journey of life-saving drugs to market. In 2018, 73% of the novel drugs approved by the U.S. Federal Drug Administration (FDA) were designated under one or more expedited development program categories (Fast Track, Breakthrough Therapy, Priority Review, and Accelerated Approval).ᶦ

Sanofi out­lines big API plans as coro­n­avirus out­break re­port­ed­ly threat­ens short­age of 150 drugs

As the world becomes increasingly dependant on Asia for the ingredients of its medicines, Sanofi sees business to be done in Europe.

The French drugmaker said it’s creating the world’s second largest active pharmaceutical ingredients (API) manufacturer by spinning out its six current sites into a standalone company: Brindisi (Italy), Frankfurt Chemistry (Germany), Haverhill (UK), St Aubin les Elbeuf (France), Újpest (Hungary) and Vertolaye (France). They have mapped out €1 billion in expected sales by 2022 and 3,100 employees for the new operations headquartered in France.

Bio­gen touts new ev­i­dence from the gene ther­a­py com­pa­ny it wa­gered $800M on

A year ago, Biogen made a big bet on a small gene therapy company. Now they have new evidence one of their therapies could work.

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Anthony Fauci (AP Images)

UP­DAT­ED: NIH-part­nered Mod­er­na ships off its PhI-ready coro­n­avirus vac­cine can­di­date to a sea of un­cer­tain­ty

Off it goes.

Moderna has shipped the first batch of its mRNA vaccine against SARS-CoV-2 from its manufacturing facility in Norwood, Massachusetts, to the National Institute of Allergy and Infectious Diseases in Bethesda, Maryland, for a pioneering Phase I study.

It’s a hectic race against time. In the 42 days since Moderna selected the sequence they would use to develop their vaccine — a record time, no less — the number of confirmed cases around the world has surged astronomically from a few dozen to over 80,000, per WHO and Johns Hopkins estimates.

The candidate that they came up with, mRNA-1273, encodes for a prefusion stabilized form of the spike protein, which gives the virus its crown shape and plays a key role in transmission. The Coalition for Epidemic Preparedness Innovations, the Oslo-based group better known as CEPI, funded the manufacture of this batch.

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In fi­nal re­port, ICER ap­pears to have a change of heart on new acute mi­graine ther­a­pies

ICER appears to have reversed course on the fresh crop of acute migraine therapies.

The cost-effectiveness watchdog in November issued a draft report suggesting that existing generic medicines are more effective and cheaper than Allergan’s December-approved CGRP ubrogepant, Biohaven rival molecule, rimegepant (which is under FDA review), and Lilly’s October-sanctioned lasmiditan, which binds to 5-HT1F receptors.

Bi­cy­cle Ther­a­peu­tics takes Roche's Genen­tech on an up to $2B im­muno-on­col­o­gy ride

Bicycle Therapeutics — which is developing a new class of chemically synthesized drugs designed to be pharmacologically as active as biologics, yet manufactured as small molecules —  has scored another big partner: Roche’s Genentech.

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When drug val­ue as­sess­ment meets re­al-world ev­i­dence: ICER en­lists Ae­tion in pric­ing eval­u­a­tion

In a union of two of the hottest trends in the US biopharma world, ICER is teaming up with a high-profile company to integrate real-world evidence in their assessment of treatment value.

The drug pricing watchdog — formally the Institute for Clinical and Economic Review — said it will utilize Aetion’s evidence platform in “select upcoming assessments” and their new 24-month re-evaluations of drugs granted accelerated approval by the FDA.

Anthony Fauci, AP Images

First US Covid-19 tri­als set to get un­der­way in Ne­bras­ka and Wash­ing­ton, backed by NIH

The first US clinical trials on the novel coronavirus are scheduled to get underway next month at the University of Nebraska Medical Center, where American passengers were taken after being evacuated from the Diamond Princess cruise ship, and at the Kaiser Permanente Washington Health Research Institute. Both trials are sponsored by the NIH’s National Institute for Allergy and Infectious Diseases, which has led the US’s medical response to the outbreak.

UP­DAT­ED: NGM Bio takes leap for­ward in crowd­ed NASH field

South San Francisco-based NGM Bio may have underwhelmed with its interim analysis of a key cohort from a mid-stage NASH study last fall — but stellar topline data unveiled on Monday showed the compound induced significant signs of antifibrotic activity, NASH resolution and liver fat reduction, sending the company’s stock soaring.

There are an estimated 50+ companies focused on developing drugs for non-alcoholic steatohepatitis, or NASH, a common liver disease that has long flummoxed researchers. The first wave of NASH drug developers struggled with efficacy as well as safety — and companies big and small have crashed and burned.

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