FDA [via Andrew Harnik/AP]

Boehringer In­gel­heim's Ofev wins FDA ap­proval for use in scle­ro­der­ma pa­tients

Five years af­ter se­cur­ing its first ap­proval in id­io­path­ic lung fi­bro­sis,  Boehringer In­gel­heim’s drug Ofev has been cleared to slow the rate of pul­monary de­cline in pa­tients with an­oth­er lung scar­ring dis­ease.

The ap­proval — for pa­tients with in­ter­sti­tial lung dis­ease as­so­ci­at­ed with sys­temic scle­ro­sis or scle­ro­der­ma (SSc-ILD) — marks the first FDA nod for the rare lung con­di­tion.

The agency’s en­dorse­ment was based on da­ta from a 576-pa­tient place­bo-con­trolled study. The main goal of the tri­al was to mea­sure lung func­tion via forced vi­tal ca­pac­i­ty (FVC) — de­fined as the amount of air that can be forcibly ex­haled from the lungs af­ter tak­ing the deep­est breath pos­si­ble — at the end of 52 weeks, al­though some pa­tients were treat­ed for up to 100 weeks.

Pa­tients giv­en the drug, known chem­i­cal­ly as nintedanib, ex­pe­ri­enced a slow­er rate of FVC de­cline. Those giv­en the place­bo saw a de­cline of 93.3 (mL/year), ver­sus 52.4 (mL/year) in pa­tients on the drug (p=0.04).

On the safe­ty side how­ev­er, there were some side-ef­fects — large­ly di­ar­rhea — that led to dose re­duc­tions. Da­ta showed 34% of Ofev-treat­ed pa­tients, com­pared to 4% of place­bo-treat­ed pa­tients, re­quired tweaks to their dos­ing.

Scle­ro­der­ma is a rare con­di­tion that is char­ac­ter­ized by the thick­en­ing and scar­ring of tis­sues — in­clud­ing the lungs — across the body. In­ter­sti­tial lung dis­ease (ILD) is a con­di­tion af­fect­ing con­nec­tive tis­sue that forms the sup­port struc­ture of the alve­oli in lungs, and is com­mon in scle­ro­der­ma. SSc-ILD, which af­fects rough­ly 50,000 Amer­i­cans, caus­es lung func­tion to pro­gres­sive­ly de­cline, and is the lead­ing cause of death in scle­ro­der­ma pa­tients.

In the first half of this year, net sales of Ofev to treat id­io­path­ic lung fi­bro­sis (IPF) rose by 21.6% to 677 mil­lion eu­ros (about $747 mil­lion) on a cur­ren­cy-ad­just­ed ba­sis.

Ofev was cleared for use in IPF in Oc­to­ber 2014 — on the same day as ri­val Roche’s Es­bri­et, kick­start­ing a scram­ble for mar­ket share.

This Ju­ly, Boehringer signed a pact with Bridge Bio­ther­a­peu­tics to ex­pand its IPF ar­se­nal. The Ko­re­an biotech has a au­to­tax­in in­hibitor, BBT-877, in ear­ly-stage de­vel­op­ment.

Mi­no­ryx and Sper­o­genix ink an ex­clu­sive li­cense agree­ment to de­vel­op and com­mer­cial­ize lerigli­ta­zone in Chi­na

September 23, 2020 – Hong Kong, Beijing, Shanghai (China) and Mataró, Barcelona (Spain)  

Minoryx will receive an upfront and milestone payments of up to $78 million, as well as double digit royalties on annual net sales 

Sperogenix will receive exclusive rights to develop and commercialize leriglitazone for the treatment of X-linked adrenoleukodystrophy (X-ALD), a rare life-threatening neurological condition

FDA chief Stephen Hahn on Capitol Hill earlier this week (Getty Images)

As FDA buck­les un­der the strain of a pan­dem­ic work­load, Trump again ac­cus­es the agency of a po­lit­i­cal hit job

Peter Marks appeared before a virtual SVB Leerink audience yesterday and said that his staff at FDA’s CBER is on the verge of working around the clock. Manufacturing inspections, policy work and sponsor communications have all been pushed down the to-do list so that they can be responsive to Covid-related interactions. And the agency’s objective right now? “To save as many lives as we can,” Marks said, likening the mortality on the current outbreak as equivalent to “a nuclear bomb on a small city.”

Daniel O'Day, Gilead CEO (Kevin Dietsch/UPI/Bloomberg via Getty Images)

Play-by-play of Gilead­'s $21B Im­munomedics buy­out de­tails a fren­zied push — and mints a new biotech bil­lion­aire

Immunomedics had not really been looking for a buyout when the year began. Excited by its BLA for Trodelvy, submitted to the FDA in late 2019, executive chairman Behzad Aghazadeh started off looking for potential licensing deals and zeroed in on four potential partners, including Gilead, following January’s JP Morgan Healthcare Conference in San Francisco. Such talks advanced throughout the year, with discussions advancing to the second round in mid-August.

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President Donald Trump reacts after signing an executive order following his remarks on his healthcare policies yesterday in Charlotte, North Carolina (Getty Images)

Op-ed: Will phar­ma re­al­ly pay for Trump’s lat­est law­less promise to 33 mil­lion Medicare ben­e­fi­cia­ries? Not like­ly

Sitting atop the executive branch, President Donald Trump is the ultimate authority at the FDA. He can fast track any vaccine to approval himself. If it came to that, of course.

What he can’t do is unilaterally order the legislative branch to loosen the Treasury’s coffers for $6.6 billion. Nor can he command pharmaceutical companies to pay for $200 vouchers sent to 33 million Medicare beneficiaries for prescription drugs before the election.

President Donald Trump and FDA Commissioner Stephen Hahn (AP Images)

FDA is­sues fi­nal rule al­low­ing im­por­ta­tion of drugs from Cana­da — but al­so keeps the pow­er to re­voke it

Just over a month away from the presidential election, the FDA has issued a final regulation fulfilling President Trump’s promise to let states import certain prescription drugs from Canada.

On Thursday, Trump told a crowd in North Carolina that the new rule goes into effect “today.” But the published regulation states that it won’t take effect for 60 days. And even then, it could be a while before cheaper drugs make it across the border.

The win­dow is wide open as four more biotechs join the go-go IPO class of 2020

It’s another day of hauling cash in the biopharma world as four more IPOs priced Friday and a fifth filed its initial paperwork.

The biggest offering comes from PMV Pharma, an oncology biotech focusing on p53 mutations, which raised $211.8 million after pricing shares at $18 apiece. Prelude Therapeutics, developing PRMT5 inhibitors for rare cancers, was next with a $158 million raise, pricing shares at $19 each. Graybug Vision raised $90 million after pricing at $16 per share for its wet AMD candidates, and breast cancer biotech Greenwich Lifesciences brought up the rear with a small, $7 million raise after pricing shares at $5.75.

J&J of­fers PhI/IIa da­ta show­ing its sin­gle-dose vac­cine can stir up suf­fi­cient im­mune re­sponse

Days after J&J dosed the first participants of its Phase III ENSEMBLE trial, the pharma giant has detailed the early-stage data that gave them confidence in a single-dose regimen.

Testing two dose levels either as a single dose or in a two-dose schedule spaced by 56 days in, the scientists from Janssen, the J&J subsidiary developing its vaccine, reported that the low dose induced a similar immune response as the high dose. The interim Phase I/IIa results were posted in a preprint on medRxiv.

New York governor Andrew Cuomo (AP Images)

An­drew Cuo­mo says New York will un­der­take its own vac­cine re­view process, and wouldn’t rec­om­mend trust­ing the fed­er­al gov­ern­ment

The concerns keep mounting over President Donald Trump’s politicization of the FDA and other federal agencies guiding the development of a safe and effective vaccine. And today, the telegenic New York governor Andrew Cuomo appeared to introduce even more politics into the matter — latest in an ongoing series of incidents that have cast the proudly independent FDA in starkly political terms.

During his daily press conference Cuomo said that the state will review any coronavirus vaccines approved by the federal government, citing a lack of trust in the Trump administration. The announcement comes one day after Trump accused the FDA of making an “extremely political” move in proposing stricter vaccine guidance.

President Trump walks past HHS secretary Alex Azar (Getty Images)

Azar falls in line un­der Trump again. Ex­perts say he's re­in­forc­ing a dark sig­nal sent to the FDA

In the latest incident where Alex Azar has steadfastly taken the side of President Donald Trump over that of the FDA, the HHS secretary was noncommittal this morning when asked if he supports the attempt by his subordinates at the FDA to strengthen guidelines for a vaccine EUA.

Appearing on NBC’s Today Show, the HHS secretary muddied the waters, stating that the guidance that matters is the one that is “actually already out there.”