FDA to re­quire cer­tain IND safe­ty re­ports be sub­mit­ted to FAERS

The FDA on Tues­day is­sued draft guid­ance re­quir­ing spon­sors to sub­mit in­ves­ti­ga­tion­al new drug (IND) safe­ty re­ports for se­ri­ous and un­ex­pect­ed sus­pect­ed ad­verse events to the FDA Ad­verse Event Re­port­ing Sys­tem (FAERS) start­ing 24 months af­ter the guid­ance is fi­nal­ized.

Cur­rent­ly, such re­ports are sub­mit­ted to the FDA in elec­tron­ic com­mon tech­ni­cal doc­u­ment (eCTD) for­mat us­ing PDF files, which the FDA says are “in­ef­fi­cient and la­bor in­ten­sive” to re­view and track.

“Sub­mis­sion of this im­por­tant safe­ty in­for­ma­tion to FAERS as struc­tured da­ta el­e­ments will im­prove the FDA’s abil­i­ty to re­view and track these po­ten­tial safe­ty sig­nals that oc­cur dur­ing the con­duct of clin­i­cal tri­als, and will pro­vide spon­sors with a re­port­ing for­mat that is con­sis­tent with In­ter­na­tion­al Coun­cil for Har­mon­i­sa­tion (ICH) guide­lines and re­port­ing re­quire­ments to oth­er reg­u­la­to­ry agen­cies,” the FDA writes.

The switch to sub­mit­ting IND safe­ty re­ports for se­ri­ous and un­ex­pect­ed sus­pect­ed ad­verse events is part of the FDA’s ef­fort to im­ple­ment the elec­tron­ic sub­mis­sion re­quire­ments of Sec­tion 745A(a) of the Fed­er­al Food, Drug, and Cos­met­ic Act.

While fed­er­al agen­cies are not typ­i­cal­ly able to es­tab­lish new re­quire­ments via guid­ance, Con­gress gave the FDA ex­plic­it au­tho­riza­tion to do so to es­tab­lish the for­mat for elec­tron­ic sub­mis­sions un­der Sec­tion 745A(a).

For now, the FDA says spon­sors will be able to con­tin­ue sub­mit­ting such IND safe­ty re­ports via eCTD, though spon­sors may vol­un­tar­i­ly sub­mit the re­ports to FAERS be­fore the re­quire­ment is in ef­fect.

Once ef­fec­tive, the guid­ance will su­per­sede the FDA’s fi­nal guid­ance Pro­vid­ing Reg­u­la­to­ry Sub­mis­sions in Elec­tron­ic For­mat — Cer­tain Hu­man Phar­ma­ceu­ti­cal Prod­uct Ap­pli­ca­tions and Re­lat­ed Sub­mis­sions Us­ing the eCTD Spec­i­fi­ca­tions for IND safe­ty re­ports that fall with­in the scope of the new guid­ance.

Un­der the pol­i­cy laid out in the guid­ance, spon­sors must sub­mit IND safe­ty re­ports to FAERS via the FDA’s Elec­tron­ic Sub­mis­sion Gate­way (ESG) or Safe­ty Re­port­ing Por­tal (SRP).

The FDA notes that the guid­ance does not ap­ply to IND safe­ty re­ports for any­thing oth­er than se­ri­ous and un­ex­pect­ed sus­pect­ed ad­verse events, such as re­ports de­tail­ing find­ings from oth­er stud­ies, find­ings from an­i­mal or in vit­ro test­ing or of in­creased rate of oc­cur­rence of se­ri­ous sus­pect­ed ad­verse re­ac­tions.

Ad­di­tion­al­ly, the FDA says that sub­mis­sions for non­com­mer­cial INDs will be ex­empt from the elec­tron­ic sub­mis­sion re­quire­ments un­der Sec­tion 745A(a), though the agency en­cour­ages such spon­sors to do so.

Along­side the draft guid­ance, the FDA is­sued a sep­a­rate tech­ni­cal con­for­mance guide meant to sup­ple­ment the draft guid­ance by de­tail­ing the for­mat that should be used for IND safe­ty re­ports sub­mit­ted to FAERS as in­di­vid­ual case safe­ty re­ports (IC­SRs) and IND safe­ty re­ports that should be sub­mit­ted to the FDA in elec­tron­ic com­mon tech­ni­cal doc­u­ment (eCTD) for­mat.

RAPS: First pub­lished in Reg­u­la­to­ry Fo­cus™ by the Reg­u­la­to­ry Af­fairs Pro­fes­sion­als So­ci­ety, the largest glob­al or­ga­ni­za­tion of and for those in­volved with the reg­u­la­tion of health­care prod­ucts. Click here for more in­for­ma­tion

Com­mu­ni­cat­ing the val­ue of pre­ci­sion med­i­cine

By Natasha Cowan, Content Marketing Manager at Blue Latitude Health.
Many stakeholders are confused by novel precision medicines, including patients and healthcare professionals. So, how can industry help them to navigate this complexity?

Precision medicine represents a new paradigm in healthcare. It embodies the shift from treating many patients with the same therapy, to having the tools to identify the best treatment for every patient.

Spe­cial re­port: Twen­ty ex­tra­or­di­nary women in bio­phar­ma R&D who worked their way to the top

What differentiates a woman leader in biopharma R&D from a man?

Not much, except there are fewer of them in senior posts. Data suggest women are not more risk-averse, family-oriented or less confident than their male counterparts — indeed the differences between the two sexes are negligible. But a glance at the top R&D positions in Big Pharma leaves little doubt that upward migration in the executive ranks of biopharma R&D is tough.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 65,000+ biopharma pros reading Endpoints daily — and it's free.

Ed­i­tas and Cel­gene sub Juno are tack­ling hottest im­munother­a­py cells

As the first CRISPR-edited cancer patients watch their treatments unfold, one of the first CRISPR companies is rejigging a major oncology deal.

Editas Medicine is amending its long-running collaboration with Celgene and their subsidiary Juno Therapeutics. The new deal will expand the focus of their work to cover a subset of immune cells that have become an increasingly hot target for immunotherapy: gamma-delta cells.

FDA Vas­cepa re­view spot­lights new safe­ty sig­nals, pos­si­ble min­er­al oil spoil­er as Amarin hunts a block­buster ap­proval

An in-house FDA review of Amarin’s Vascepa raises a set of hurdles the biotech will have to clear if the biotech expects to get the long-awaited FDA approval that could set it on a path to superstar status. But it appears that Amarin has survived another potential setback without introducing a major new threat to its prospects.
The stakes don’t get much higher, with analysts saying a win this week for Amarin could lead to billions in new sales — provided the agency stamps it with an OK. And investors liked what they say in the FDA review this morning, bumping the stock $AMRN 17%.
The insider take at the agency includes a note on two new safety signals seen in the big cardio outcomes study of the omega-3 fatty acid drug that shocked many analysts with a solid set of efficacy data. There’s a key concern over whether the use of mineral oil in the placebo skewed LDL levels in such a way that tilted the data in Amarin’s favor.
The FDA overview was written by John Sharretts, the acting deputy director in the Division of Metabolism and Endocrinology Products. 
On the safety side, the internal review focused on a 3.1% versus 2.1% rate of adjudicated events of atrial fibrillation or atrial flutter requiring hospitalization. But they also say a-fib shouldn’t confound the benefit-safety of the drug — given the improvement on MACE — or prevent its use. And then there was also a higher rate of bleeding events in the drug arm.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 65,000+ biopharma pros reading Endpoints daily — and it's free.

Prakash Raman. Flagship

Flag­ship woos No­var­tis top deal­mak­er Prakash Ra­man in move to get the BD ball rolling ear­ly

Flagship Pioneering likes to be ahead of its times — so far ahead, perhaps, that it is often challenging to find partners for their startups while the scientific scaffolding is underway. But Prakash Raman is here to change that.

Raman, who most recently headed up business development at the Novartis Institutes for BioMedical Research, became Flagship’s first chief business development officer two weeks ago. By acting as a “central resource” for the 100 companies in the venture fund’s portfolio, he hopes to help entrepreneurs and management teams strategize about dealmaking to capture value beyond the near-term validation of their platform technologies, Raman told Endpoints News.

FDA puts Sol­id Bio’s lead gene ther­a­py pro­gram on hold — again — af­ter an­oth­er pa­tient is hurt by SGT-001

Solid Biosciences continues to be plagued by safety issues.

Close to 18 months after the gene therapy biotech was able to quickly shed an FDA hold on their lead Duchenne muscular dystrophy program for SGT-001, regulators have stepped back in to force another halt after another patient was hit hard by a set of serious adverse events remarkably similar to the first set.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 65,000+ biopharma pros reading Endpoints daily — and it's free.

Bill Haney, Skyhawk

Cel­gene ex­ecs shell out $92M cash for a pair of R&D deals that will fit per­fect­ly in their new home at Bris­tol-My­ers

With Bristol-Myers Squibb’s Celgene buyout all but complete, the BD teams are working in perfect synchrony now. The Celgene side is going back to Skyhawk, a darling of the crowd that set out to drug RNA, and they’re adding a suite of new programs that mesh perfectly with the new regime in charge.

Celgene is shelling out $80 million in a cash upfront to add oncology, immuno-oncology and autoimmune diseases to the initial roundup of neurological targets mapped early in Skyhawk’s existence.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 65,000+ biopharma pros reading Endpoints daily — and it's free.

Reata's bar­dox­olone of­fers promise in pa­tients with rare kid­ney dis­or­der

After surprising Wall Street with positive data on its drug, omaveloxolone, in patients suffering from a notoriously hard-to-treat degenerative neuromuscular disorder last month, Reata Pharma on Monday unveiled pivotal results from a trial testing another drug, bardoxolone, in patients with a rare, genetic form of chronic kidney disease for which there exist no approved therapies.

Bardoxolone, like Reata’s other lead drug — omaveloxolone — is a small molecule engineered to bind to a gene called Keap1 to enhance the activity of the protein Nrf2 in order to defuse inflammation.

Am­gen ax­es 149 of its staff in Cam­bridge of­fice; Evotec, Mil­li­pore­Sig­ma en­ter re­search pact

→ Amgen has submitted a Worker Adjustment and Retraining Act (WARN) — a warning of impending mass layoffs 60 days in advance of the date — to the state of Massachusetts in the wake of the company’s exodus from the neurosciences R&D sector. David Reese, the company’s R&D chief, said at the time that the company is cutting ties in the field to focus on other undisclosed areas. In its WARN notice, the Cambridge-based company stated that 149 of its employees would be affected — among the total 180 being let go. The terminations will take effect on December 31, 2019.