Whether it be for an avid
investor or for an occasional follower of the pharmaceutical industry,
significant FDA events pose some of the greatest interest, and act as some of
the most explosive catalysts of change in a company’s share price. For a small
biotech located anywhere in the world, a marketing approval granted by the FDA
is arguably the most contested prize in the industry.
As a result of the 2009 – 2015
Patent Cliff, industry observers can expect increased crowding on the FDA
calendar, as many new drugs are currently being developed to offset cliff
losses. 2012 was one of the record years for drugs reaching the market, with 38
new medicines approved by the FDA, a new 16 year record. 2013 appears equally
promising, with 22 scheduled FDA decisions so far – half of which are
anticipated in the first quarter.
The vibrancy of the biotech
industry is reflected in the performance of the NASDAQ biotechnology index,
which, having returned
~35% growth in 2012 (more than 4 times that of the Dow Jones), continues to
soar above the other indices (fig. 1).
On the FDA calendar are numerous
events ranging from Investigational New Drug (IND) applications down to the
very final stage of a New Drug Application (NDA) or a Biologics License
Application (BLA) which, if successful, grants a new medicine approval to be
marketed and sold in the US – the world's largest pharmaceutical market. The
date by which the FDA aims to deliver its marketing verdict for an NDA or BLA is
called the Prescription
Drug User Fee Act (PDUFA) date, following which the applicant may be granted or
refused marketing approval, or requested to provide additional documentation
and/or conduct further studies.
The PDUFA date carries crucial importance
as it can signify the birth or death of a new medicine. To a pharma and biotech
investor, an FDA marketing decision can potentially crash or soar a stock. PDUFA
dates normally carry a significant stock runup up to the date, and PDUFA-related
intraday surges of up to 300% are not unheard of, which should be incentive
enough to closely follow, and understand, the FDA calendar.
Q1 2013: Scheduled Drug Approvals Overview
The next three months (January
through March of 2013) anticipate a more-than-usually saturated FDA calendar,
with a total of 11 PDUFA dates (table 1). Salix Pharmaceuticals and Santarus
Pharma kicked off this year’s marketing approval list with the anti-diarrhoeal
drug Fulyzaq and the Ulcerative Colitis treatment UCERIS, which was
granted approval two days ahead of schedule. 6 drugs are still lined up to face
FDA’s advisory panel this quarter.
Table 1. Scheduled PDUFA dates on the FDA calendar; January
– March 2013
|
Date
|
Company
(NASDAQ Ticker)
|
Drug
|
Indication
|
||
|
Brand Name
|
Generic Name
|
||||
|
Jan 2
|
Salix Pharmaceuticals (SLXP)
|
Fulyzaq
|
Crofelemer
|
Diarrhea associated with anti-HIV drugs such as nucleoside
analog reverse transcriptase inhibitors and protease
inhibitors – FDA approval granted
|
|
|
Jan 14
|
Santarus
(SNTS)
|
UCERIS
|
Budesonide
(novel formulation)
|
Induction of remission in patients with active, mild
to moderate Ulcerative Colitis –FDA approval granted
|
|
|
Jan 17
|
NuPathe
(PATH)
|
Zecurit
(Zelrix)
|
Sumatriptan
(novel delivery) |
Migraine Patch –FDA approval granted
|
|
|
Jan 21
|
Rytary (IPX066)
|
Carbidopa and Levodopa
(extended release)
|
Idiopathic Parkinson’s Disease (PD) - received a
complete response letter
|
||
|
Jan 23
|
Hyperion Therapeutics
(HPTX)
|
Ravicti
|
Glycerol phenylbutyrate
|
Urea Cycle Disorders –FDA approval granted
|
|
|
Jan 29
|
Isis Pharmaceuticals,
(ISIS), Genzyme
|
KYNAMRO
|
Mipomersen sodium
|
Homozygous Familial Hypercholesterolemia
|
|
|
Feb 2
|
Ampligen®
|
Rintatolimod
|
Double stranded RNA drug for Chronic Fatigue Syndrome (CFS)
|
||
|
Feb 24
|
Dynavax Technologies
(DVAX)
|
HEPLISAV
|
Hepatitis B vaccine
|
Biologic intended for immunization against infection
caused by all known subtypes of hepatitis B virus
|
|
|
Feb 26
|
ImmunoGen (IMGN)
Roche
|
T-DM1
|
Trastuzumab emtansine
|
HER2+ Metastatic Breast Cancer (mBC)
|
|
|
Mar 1
|
Zogenix (ZGNX)
|
Zohydro ER
|
Hydrocodone bitartrate
(extended release)
|
Moderate to severe chronic pain
|
|
|
Mar 27
|
A.P. Pharma
(APPA)
|
APF530
|
Granisetron (sustained release)
|
Acute- and delayed-onset chemotherapy-induced nausea and vomiting
(CINV)
|
|
It is common for the FDA to announce 3-month PDUFA delays,
and several of the drugs awaiting a verdict have already undergone such
extensions. Statistically, only 35% of new drug applications receive FDA
approval in the first review cycle[i].
It is likely that a number of drugs on the Q1-2013 calendar will face similar delays,
partly due to increased application volume this quarter.
Therapeutic landscape
Just
over half of the drugs (6 out of 11) on the FDA calendar this quarter are novel
chemical or biological entities, whilst the rest are non-innovative
reformulations and repositioning of formerly approved drugs, combination
therapies, etc. In terms of therapeutic area, nearly a third
of NDAs in Q1 adress Central Nervous System (CNS) disorders, followed by
gastrointestinal and autoimmune/inflammatory targets (fig. 2). Only one of the
drugs, Dynavax’s HEPLISAV vaccine, is a biologic.
Figure 2. Drugs awaiting FDA marketing
decisions in Q1- 2013, by therapeutic area
Market capitalization
Interestingly,
the Q1 2013 FDA calendar does not feature Big Pharma players, with the
exception of those which have partnered with the drug developers covered in
this report. In what has become the standard prior to FDA applications, all of
the companies featured in this Q1 2013 report are public. The majority have a
market capitalization of US$ 2 billion or less (fig. 3), with only one mid-cap
company – Salix Pharmaceuticals, worth US$ 2.69 billion.
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[i] http://www.topra.org/sites/default/files/assets/pdf/f_houn_17_may_2011_topra_nj.pdf
