Archive for October 2010

FDA approves blood indication for Sprycel

Today the FDA approved a new indication for Bristol-Myers Squibb‘s Sprycel (dasatinib) for the treatment of a rare blood cancer when it is first diagnosed. The cancer, called Philadelphia chromosome positive chronic phase chronic myeloid leukemia (Ph+ CP-CML), is a slowly progressing blood and bone marrow disease linked to a genetic abnormality.

BYDUREON rejection

Nobody saw this one coming!  Nobody but the FDA that is.  Cardiovascular safety has been a very hot item with the FDA for a very long time.  The rejection of the BYDUREON NDA certainly sends a message to the rest of the industry on two points.  First, the cardiovascular profile has still got  to be extremely clean. Nothing new there.  We’ve seen several high profile drugs rejected this year because of CV safety issues.   Second, if the product is an extended release, the pharmacokinetics have got to match the dosing regimen.  Nothing new there either.  Combine these two points such that you get some cardiovascular safety issues that might be associated with drug accumulation and it spells rejection.

FDA approves Pradaxa to prevent stroke in people with atrial fibrillation

On Tuesday, the FDA approved Boehringer Ingelheim’s Pradaxa capsules (dabigatran etexilate) for the prevention of stroke and blood clots in patients with abnormal heart rhythm (atrial fibrillation).

Alexza-Staccato lorazepine PDUFA Date follow up

Alexza announced today (Oct 11, 2010) that they have received a Complete Response letter from the FDA.  That they did not get an approval is not a surprise (see our earlier opinion).  It’s also not a surprise to us that one of the concerns that the FDA has regards the delivery device, a new device that has yet to be approved for use with any drug. What is a surprise is the safety issue raised by the FDA regarding pulmonary function tests from 3 Phase I safety studies.  Yes, Phase 1 studies.  Why wasn’t this addressed at the End of Phase 2 meeting?

XYREM Update

We’ve noted in previous postings the failure of JAZZ to convince the FDA Advisory Committee that XYREM should be approved for fibromyalgia, and our opinion that the FDA is unlikely to approve the drug as the PDUFA Date approaches.  Motley’s Brian Orelli seems to share our opinion, as do the vast majority of the people responding to his poll.  It does seem meaningful to restate our position at this time, both in support of Mr. Orelli, but also to clarify the regulatory position as we see it.  There were significant issues raised about the RISK associated with this product.  The REMS program offered by XYREM was felt to be inadequate by the Advisory Committee.  More importantly, whatever the drug does or does not do in the intended patient population, in the absence of an adequate REMS program, it increases the risk of the non-patient population with no benefit to these non-patients. The FDA mandate is to protect and promote the health of the US population.  With an inadequate REMS, the FDA must protect and not approve this drug until an adequate REMS is in place.


October 11, 2010 is the PDUFA Date for Jazz Pharmaceuticals’ (JAZZ) XYREM for the fibromyalgia indication.  The drug is currently approved for narcolepsy.  Jazz  presented the XYREM fibromyalgia data to the FDA Advisory Committee on August 20, 2010 and did not receive a positive recommendation from the Committee.  The Committee rejected their REMS proposal 20-2.  It is unlikely the deficiencies in the REMS will be overcome by October 11, 2010.

PDUFA Date – STACCATO loxapine

October 11, 2010 is the PDUFA Date for Alexza Pharmaceuticals STACCATO loxapine, an inhalation formulation of loxapine intended for rapid treatment of schizophrenia and bipolar disorders.  The company reports that this is the first NDA that it has submitted using the STACCATO inhalation technology.  Phase III clinical studies are reported as having been submitted that support both indications but few details are available other than reports that both primary and secondary endpoints were met.  Getting approval for both indications using new technology seems like a bit of a hurdle for a company that is submitting its first NDA.


October 3, 2010 is the scheduled PDUFA Date for Javelin Pharmaceuticals DYLOJECT for the management of post operative pain.  The company has been silent on any actions that may have been taking place with the FDA on this submission.  One possible source of a delay on the approval of this application could be gaining assurance that the problems that led to the withdrawal of the product in the UK earlier this year are not potential problems in the US.


October 4, 2010 is the PDUFA Date for the Human Genome Sciences product ZALBIN for hepititis C.  We’ve not seen anything since the company announced that it had received a Discipline Review letter from the FDA on June 6, 2010.  The FDA cited Benefit/Risk issues for the dosing regimen.  The company indicated at that time that they thought licensure was unlikely.