CDER was fast off the starting blocks in 2012, approving five novel agents in January – including a one-two punch of two with the shortest approval times in recent agency history on the last two days of the month.

The January 2012 novel approvals are dominated by oncology products (4 of 5), priority reviews (3 of 5) and small molecules (4 NMEs vs. 1 therapeutic BLA). CBER has not yet posted a novel approval.

Vertex Pharmaceuticals Inc.’s Kalydeco (ivacaftor), the first disease-modifying therapy for cystic fibrosis, entered the record books on Jan. 31 with the shortest time to approval for a non-oncology therapeutic in the contemporary era: 3.4 months from NDA submission to marketing clearance. The small review period is matched by the small population eligible for the orphan drug, which is approved for patients with a specific mutation in the CFTR gene – about 4% of the CF population Also see "Early Success For Kalydeco Is Just The Beginning For Vertex’s CF Drug" - Pink Sheet, 6 February, 2012..

The day before Kalydeco’s approval, FDA cleared the first Hedgehog pathway inhibitor, Genentech Inc./Curis Inc.’s Erivedge (vismodegib), 4.7 months after the NDA for treatment of advanced basal cell carcinoma was submitted Also see "Curis Set For Transformative Year With Erivedge Skin Cancer Approval" - Pink Sheet, 6 February, 2012.. Another agent for cancer patients, BTG International Inc.’s orphan methotrexate toxicity antidote Voraxaze (glucarpidase), saw a first-cycle priority review clearance on a more conventional timeframe, receiving approval one day before its user fee goal Also see "BTG Gains First U.S. Approval With Methotrexate Antidote Voraxaze" - Pink Sheet, 18 January, 2012..

Sometimes, A Need For Speed: The 12 Fastest Approvals Of The Decade
Year	Product & Sponsor	Category	Time to Approval
2001	Novartis’ Gleevec (imatinib)	Oncology (orphan population; personalized medicine indication)	2.4 mo.
2010	Sanofi’s Jevtana (cabazitaxel)	Oncology	2.6 mo.
2005	GlaxoSmithKline Fluarix (influenza virus vaccine)*	Prophylactic vaccine	3.1 mo.
2012	Vertex Pharmaceuticals’ Kalydeco (ivacaftor)	Cystic fibrosis (orphan; personalized medicine)	3.4 mo.
2004	Hameln Pharmaceuticals GmbH’s pentetate calcium trisodium & pentetate zinc trisodium**	Radiation contamination (orphan)	3.5 mo.
2003	Millennium Pharmaceuticals’ Velcade (bortezomib)	Oncology (orphan)	3.7 mo.
2004	OSI Pharmaceuticals’ Tarceva (erlotinib)	Oncology (personalized medicine)	3.7 mo.
2011	Roche’s Zelboraf (vemurafenib)	Oncology (orphan; personalized medicine)	4.3 mo.
2011	Johnson & Johnson’s Zytiga (abiraterone)	Oncology	4.3 mo.
2004	Celgene’s Vidaza (azacitidine)	Oncology (orphan)	4.7 mo.
2012	Roche/Curis’ Erivedge (vismodegib)	Oncology	4.7 mo.
2011	Pfizer’s Xalkori (crizotinib)	Oncology (orphan; personalized medicine)	4.9 mo.
Severe shortages of seasonal influenza vaccine enabled Fluarix’ clearance under accelerated approval provisions. *While FDA coded the approvals a “1,” or NME, the applications were submitted under the 505(b)(2) pathway – Ca-DTPA had been used since 1958 and Zn-DTPA since 1976 for accidents at nuclear facilities. Hameln has discontinued marketing the radiation contamination products.

Leo Pharma AS’ topical actinic keratosis treatment Picato (ingenol) offers a convenience advantage over other topical therapies for AK, but the drug, which induces cell death, received an on-time standard review Also see "LEO Plays Up Rapid Action Of Picato For Actinic Keratosis" - Pink Sheet, 26 January, 2012..

Pfizer Inc. also received approval on the standard 10-month review PDUFA goal timeframe for its second-generation VEGF inhibitor Inlyta (axitinib) for advanced renal cell carcinoma after the failure of one prior systemic therapy Also see "Inlyta Approval Expands Pfizer Kidney Cancer Options, But With Unique Safety Profile" - Pink Sheet, 27 January, 2012.. RCC has been a very active field for NMEs – Inlyta is Pfizer’s third RCC agent – and FDA, in its Jan. 27 announcement of the axitinib approval, quoted Office of Hematology and Oncology Products Director Richard Pazdur on the “unprecedented level of drug development” that has “significantly altered the treatment paradigm of metastatic kidney cancer.”

Momentous Diabetes Drug Decisions

One of FDA’s biggest approval decisions in January was for a product that is not an NME. The agency finally cleared Amylin Pharmaceuticals Inc./Alkermes PLC’s Bydureon (exenatide extended-release), a once-weekly formulation of Amylin’s GLP-1 analog Byetta, on Jan. 27, almost 32 months and two “complete response” letters after NDA submission Also see "With Bydureon’s U.S. Approval, Amylin Extends Exenatide Franchise" - Pink Sheet, 27 January, 2012.. Bydureon’s convenience advantage is expected to challenge the leading GLP-1 anti-diabetic, Novo Nordisk Inc.’s blockbuster Victoza Also see "Amylin’s Bydureon Approval: How Does It Sit With Novo Nordisk?" - Pink Sheet, 6 February, 2012.

FDA dealt a big setback to Bristol-Myers Squibb Co. and AstraZeneca PLC’s chances for a 2012 NME when it issued a “complete response” letter Jan. 17 for dapagliflozin, a first-in-class sodium-dependent glucose co-transporter-2 inhibitor, also for type 2 diabetes. Bristol signaled that the concerns FDA expressed in the letter could necessitate further clinical trials – and delays – for the drug Also see "Dapagliflozin Faces Ominous Prospect Of New Trials" - Pink Sheet, 19 January, 2012..

CDER Approvals In January Came Early And Often

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