Af­ter two years of ne­go­ti­a­tions, FDA gets its user fee pro­grams au­tho­rized through 2022

Pres­i­dent Don­ald Trump signed the FDA Reau­tho­riza­tion Act of 2017 in­to law late Fri­day, qui­et­ly end­ing a two-year long process that un­til sev­er­al weeks ago threat­ened to dis­rupt agency op­er­a­tions.

Al­though the leg­is­la­tion was passed by both hous­es of Con­gress with over­whelm­ing bi­par­ti­san sup­port — Sen­a­tor Bernie Sanders was the on­ly reg­is­tered no vote — it faced a num­ber of threats, in­clud­ing the White House’s clear de­sire to fund the FDA en­tire­ly with user fees from the com­pa­nies the agency reg­u­lates and a sen­a­tor’s long stand­ing cru­sade to un­der­cut the agency with a sweep­ing new “right-to-try” di­rec­tive.

The FDA is ex­pect­ed to col­lect $9 bil­lion in fees — $8 bil­lion for pre­scrip­tion drugs and $1 bil­lion for de­vices — be­tween 2018 and 2022, based on the fee lev­el set in the Sen­ate bill.

This marks the sixth it­er­a­tion of the PDU­FA pro­gram and the sec­ond for GDU­FA, which gov­erns gener­ic drug pro­grams. GDU­FA II cre­ates a new user fee struc­ture aimed at faster re­views and more in­ter­ac­tion be­tween the agency and gener­ics de­vel­op­ers work­ing on com­plex mol­e­cules. It comes on the heels of the FDA’s new record-set­ting pace on gener­ics ap­provals, spurred by new com­mis­sion­er Scott Got­tlieb.

Greg Walden (R-OR), the Re­pub­li­can chair­man of the House En­er­gy & Com­merce com­mit­tee had this to say on Medi­um: “FDARA is a win for pa­tients and the mil­lions of Amer­i­cans work­ing to cre­ate new cures and ther­a­pies. The bill is com­posed of four in­di­vid­ual user fee pro­grams, that pack­aged to­geth­er will de­liv­er re­sults for pa­tients and en­sure we re­main the glob­al leader in med­ical in­no­va­tion.”


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.

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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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