Pharming Receives IND Approval for Recombinant Human C1 Inhibitor From FDA
Tuesday December 21, 10:00 am ET
Company Expands Clinical Development of Product to the US
LEIDEN, The Netherlands, December 21 /PRNewswire-FirstCall/ -- Pharming Group N.V. ("Pharming" or "the Company") (Euronext: PHARM) (Amsterdam: PHAR.AS - News; OTC: PHGUF - News) announced today that the US Food and Drug Administration (FDA) has approved the Company's investigational new drug ("IND") application for recombinant human C1 inhibitor ("rhC1INH"). Based on this approval, the Company has expanded the clinical development of rhC1INH for the treatment of hereditary angioedema to the United States.
Pharming will assess the safety and efficacy of rhC1INH to treat acute attacks of hereditary angioedema ("HAE") in its US clinical trials. The clinical protocol involves a randomized, double blind, placebo controlled trial for rhC1INH in HAE patients to be conducted at multiple centers throughout the US. The Company will test rhC1INH in patients with acute attacks of HAE with two doses of rhC1INH.
"The use of rhC1INH as a replacement therapy may offer significant treatment benefits for HAE patients as the product addresses the underlying genetic basis of the disease, namely a shortage of C1 inhibitor," said Dr. Jan Nuijens, Senior Director of Clinical Development at Pharming.
"We look forward to working with investigators and patients in the United States participating in our clinical trials with rhC1INH."
In clinical studies in Europe, HAE patients treated with rhC1INH show a significant decrease in time to beginning of relief and time to complete resolution with no adverse effects reported to date.
"We are excited to receive the FDA's approval of the rhC1INH IND and to expand our clinical trials to the United States, which represents the largest single market in the world," said Dr. Francis Pinto, CEO of Pharming. "The approval of the IND underscores the potential value of rhC1INH for the treatment of HAE."
Pharming is currently conducting a randomized, double blind, placebo controlled Phase III clinical trial for rhC1INH across Europe and remains on track to file for approval of the product with the European Agency for the Evaluation of Medical Products ("EMEA") in 2005.
Background on Investigation New Drug ("IND") Application
An IND application is a request for authorization from the US Food and Drug Administration (FDA) to administer an investigational drug or biological product to humans consistent with an approved protocol. Such authorization must be secured prior to interstate shipment and administration of any new drug or biological product. The IND application contains information on the product's preclinical and clinical results, manufacturing data, and detailed clinical protocols for proposed clinical studies. An IND approval confirms that the FDA agrees that the product can be tested in humans to collect information pertinent to the products safety and efficacy.
Source: Pharming Group
Angieneurotic, the Dutch investors don't have the details on what will be included in the US trials by Pharming either. What we do know is that Pharming has been preparing these trials for over a year and that they have indicated already quite some time ago that they had everything in place in the centers where they were going to carry them out, but that they had to wait for the FDA to give the green light.
The reason why some Dutch investors have been reacting here, is that Henri Blair never even mentioned Pharming as a competitor for a HAE solution in his Conference Calls, even though they have been competitors in Europe for a number of years. I personally have been listening to his CCs for a number of years and cannot say that the information given to the Dyax shareholders has been always that objective. But I guess that is part of business. In any case, the names of Pharming and Jerini are now in the open, so no way Blair can get around the issue of competition. Obviously, he will downplay the competition to please the shareholders.
What I do wonder is, whether patients can be enrolled in clinical trials, who are using steroids. I understood from Dyax'last CC that this is the case and seriously troubling the trials (softer attacks and few retreated patients). But doesn't this also flaw the effectiveness and safety data?
This is fun!
First, neither my sister or I have an investment interest in either Pharming or Dyax. We only want her to finally have a drug that is effective for stopping attacks. The objective here was to offer some analysis of the situation. So far, just about everything posted by the Dutch guys is based on company hype. We have done quite a bit of independent research that inculdes numerous conversations and emails with key US HAE Association members who keep up on all this, and contacts with doctors/scientists that the HAE Association referred us to.
I am very sorry to say that I think you are all deluding yourself if you believe there is any way Pharming can get quick US approval of a novel recombinant product (derived from an animal) that has been given to so few humans.
The stuff I posted about Pharming's European trials not being placebo controlled came from a reliable source close to these trials. I will ask my HAE patient sister to go back and verify what she was told in an email from a prominent scientist in Europe.
One poster said patients buying C1INH would love to have the rC1INH product. That is very true, and patients would like access to any drug that can stop attacks. However, I saw an email from the HAE patient group and very, very few people (maybe 10 to 15) are buying C1INH because it is so expensive and not covered by insurance. In addition, most HAE patients can stop attacks by taking high androgen doses, and, while not pleasant, those medicines are covered by insurance. Finally, keep in mind that there just aren't all that many HAE patients out there!
There are three issues that the Pharming investors should be exploring carefully.
1. My sister says there will be four US HAE trials starting in the first quarter of 2005. She belongs to the US HAE Association and says they are very concerned that there are nowhere near enough patients to support all of these trials at the same time.
2. Look at how long it took Dyax to complete their first 48 patient US trial--well over two years. You should not neglect the difficulty of recruiting doctors and patients for a rare disease particularly now that Dyax is going to do a phase two study and the HAE Assocation says two others besides Pharming are going to do trials.
3. The effectiveness and safety of Pharming's product will have to be proved in phase 2 and phase 3 trials. And "weinrederberry" can say what he will about what Pharming has been "authorized", but my sister has talked to two US doctors who were involved in "international" trials for getting immune deficiency disease blood products approved. They say that the FDA will only accept European data for validating safety, and want US data for effectiveness. Even if the European studies were placebo controlled (and I am quite sure because of obvious ethical concerns they are not), clinical trial experts will tell you that FDA looks at foreign data as only providing support.
Frankly, we only wish that Pharming did not have so many hurdles to overcome for US approval. My sister says that many doctors "in the know" believe that until proven otherwise, C1INH will always be the best drug for treating HAE. rC1INH has obvious viral safety advantages, and would eliminate worry about plasma shortages which are always a concern with blood products.
In the end, we shall see who is right; for the good of my sister and other people who have this condition, I hope that we (and the reliable sources we have been consulting on all this) are proved wrong.
Two things about rhC1inh as compared to DX-88. Firts of all, rhC1inh is simply replacing the protein that is missing in the body, so few side effects are to be expected. It is practically identical to plasma based C1inh, which has proven it has no side effects. DX-88 is working at another level (kallikrein) in the cascade and therefore the probability of side effects is much higher. Hence many more patients to be treated by Dyax than by Pharming.
Secondly, also in the US there are plenty of patients already using plasma derived C1inh. They probably will be happy to participate in rhC1inh trials and be willing to take the risk to receive a placebo.
>>I admit that (I?) don't know anything about the design of the Pharming trials in Europe ...
Okay, I'll be back when you know.
>> ... Pharming press releases ...
Part of the homework ;-)
Merry Xmas OM
The Pharming shills do not seem to be capable of doing some basic homework. Hint, someone should ask Dr. Pinto how many people have been treated so far with the rC1INH. Then, an informed investor would speak to some experts in US drug regulation to get an idea of how many infusions the FDA might require before licensing a new drug. I will not even mention that I cannot find anything definitive on how Pharming has chosen a dose. These are just some glaring items; I have many, many more questions.
Our friends at Dyax do not have to worry about Pharming getting to market first. It just cannot and will not happen.
These are just some glaring items; I have many, many more questions.
how glad i am to help you out!
Tel: +31 (0)71 5247 400
Fax: +31 (0)71 5247 445
Hint: THEN ask Dr Blair for comment.
sleep tight, don,t let the bugs bite.
Our friends at Dyax do not have to worry about Pharming getting to market first. It just cannot and will not happen
Mr neurotic: just do your home-work. They have treated several patients in AMC (Amsterdam) with 100% results. Phase III will end in 2005 in Europe and the first patients will be treated in 2005. USA will come shortly after that!! See you later with Dyax:)