Pharmapodia Introduces Aagenaes Syndrome

It took me a bit of time to actually write this post due to limited time but here it is. 

The first orphan disease: aagenaes syndrome

Aegenaes Syndrome is a genetic disorder (proposed to be an autosomal recessive) that was first observed in 16 patients from southwest of Norway in 1968 (1).  Although it was first found in Norwegian, incidences have also been observed in other groups(2,3). 

Patients with Aagenaes syndrome often have neonatal cholestasis.  While neonatal cholestasis often disappears after some time, it recurs later on in life. The number of recurrences varies (1,4).  Overtime, as patients grow older, they will eventually develop edema at the legs and other area of the body.  Patients often experienced haemorrhages, rickets, anemia, and growth retardation.  Deaths have

Teva received marketing authorization for Zoely

Teva announced that the European Medicine Agency has approved the marketing authorization application submitted for Zoely a combination oral contraceptive product in Europe.

The company has marketing rights in France,

Takeda and Amylin discontinue the development of obesity drug

Takeda and Amylin announced that it is to discontinue the development of the combination product containing  pramlintide/metreleptin for the treatment of obesity after the companies completed their commercial reassessment of the program.  The combination product was studied in the phase II trial which was suspended due to antibody-related issues.

Although the companies discontinue the development of this

Mylan announced that the US PTO intends to reissue two patents related to Dey's Perforomist

Mylan announced that the US Patent and Trademark Office intends to reissue both of the patents related to Perforomist or formoterol fumarate inhalation solution 20 mcg/2mL vial to Dey Pharma which is a subsidiary of Mylan.

Dey Pharma issued a statement saying that Sunovion's Brovana Product which is arformoterol tartrate solution for the maintenance treatment of Chronic Obstructive Pulmonary Disease has infringed seven patents belong to Dey Pharma. 

Dey Pharma also believed that Teva's formoterol fumarate

Radient Pharmaceuticals signed agreement with Uni Pharma

Radient pharmaceuticals announced that it has entered into a distribution agreement with Uni Pharma to allow Uni Pharma import, market and sell Onko-Sure(R) in Taiwan.

Onko-Sure has been approved in in Taiwan as a simple, non-invasive cancer blood test.

Onko-Sure has also been approved in the US as a tool to monitor colorectal cancer during the treatment and for post-treatment recurrence monitoring.

The product has also been approved in Canada for lung cancer detection, treatment and recurrence monitoring.

Thinking about where it has also been approved? It has been approved in some Asian countries as well as countries in the European Union.

The US FDA put a clinical hold on Amikace's phase III trials

Insmed announced that the FDA has put a clinical hold on the company's phase III trials evaluating the use of Arikace or liposomal amikacin for inhalation in cystic fibrosis patients with pseudomonas lung infections and patients with non-tuberculous mycobacterial lung disease.

The clinical hold was issued due to the initial review of the interim results of a long term rate inhalation carcinogenicity study submitted to the FDA by Insmed. 

The FDA requires Insmed to provide additional data on Arikace as well as data from the rat study.  The company plans to cooperate and submit the required documents in the next 30 days.

The company announced that no patient has been dosed in the pending trials and enrollment as well as site initiation have been suspended after the clinical hold was issued. 

Let's brush up some information

Apnex Medical received approval to conduct the pivotal trial for its Apnex HGNS system

Apnex Medical announced that the FDA has approved the design of the company's pivotal clinical study evaluating the effectiveness of its Hypoglossal Nerve stimulation System for the treatment of obstructive sleep apnea.

The trial will be a randomized, multi-center clinical trial with multiple sites located in the US, Europe, and Australia.  It is set out to demonstrate the safety and effectiveness of the HGNS therapy in patients with moderate to severe OSA.

So what is the Apnex HGNS System exactly?

The FDA approved first specific treatment for Scorpio Sting! Welcome Anascorp!

Sometimes I wish there is a specific treatment for mosquito bites. I don't about you but every time I got a mosquito bite, it just lasts for weeks.  In addition, they also return once in a while.  Almost like an allergic reaction.

But if you happen to be in a place where the population of scorpions is high, worry no more! the US FDA has just approved a treatment for a scorpion sting by Centrurioides scorpions.  It's kind of a surprise for me to write this but yes there is a specific treatment now available in the US.The product called Anascorp will be distributed by Accredo Health Group.  It is licensed to rare Disease Therapeutics Inc of Tennessee.


The new treatment is called Anascorp which is the centrurioides (scorpion) immune F(ab')2 (Equine) injection.  The product is made from the plasma of horses that have been immunized with scorpion venom.  If a patient is sensitive to horse protein, the injection can cause early or delayed allergic reactions.

The approval cam from the data from a randomized,double-blind, placebo-controlled trial conducted in 15 children who have shown neurological signs of scorpion stings.  Treatment was given to eight subjects who were randomized into the treatment groups and resolution was observed within four hours.  In seven children who were randomized into the placebo group, only one child showed resolution of neurological signs.

The common side effects often experienced from the injection was vomiting, fever, rash, nausea, itchiness, headache, runny nose, and muscle pain.

Due to to the number of patients who are affected by the disease, Anascorp was considered an orphan drug and was approved under priority review process.

So here you go, if you get stung by a scorpion, make sure to go to the hospital who has Anascorp in formulary.  :)

The product is manufactured by Instittuto Bioclon SA de CV of Tlalpan, Mexico, D.F. 

Vosaroxin of Sunesis got US patent for method of use

Sunesis Pharmaceuticals announced that the US Patent and Trademark Office has issued a US Patent Number 7,989,468 for the methods of use for the Company's lead drug candidate vosaroxin which is being investigated for the treatment of leukemia. 

Sunesis submitted the patent application on March 14, 2005. The company has submitted patent applications in major market throughout the world including Europe, Japan, Australia and Canada.

Vosaroxin is being studied in a phase III, multinational, randomized,

Arena and Eisai update on lorcaserin

Arena and Eisai announced that it has completed the study measuring the concentration of lorcaserin in human cerebrospinal fluid and plasma in addition to related data analyses. 

Previously, brain astrocytoma (a fancy name of one of the kind of brain cancer) in rats was observed.  As a result, the companies conducted the study to determine whether this information is pertinent or applied in human.

The study was conducted in nine healthy obese volumteers.  Lorcaserin 10 mg BID was given for seven days.   The concentration in the CSF of lorcaserin was obtained on the 7th day through lumbar puncture and the concentration in the plasma of lorcaserin was obtained through blown drawn.  The concentrations were serially collected over a 12-hour period.

The company found that

• in rats, the concentration of lorcaserin in the CSF was 24 times more than its concentration in the plasma
• in human, the concentration of lorcaserin in the CSF was 1.7 times more than its concentration in the plasm

The company plans to submit the results in addition to data in other ongoing studies to the FDA as part of its process to gain the FDA approval for the use of lorcaserin in the treatment of obesity.

Besides the presence of brain astrocytoma in rats, the FDA also would like more information about mammary adenocarcinoma in female rats with other issues not clarified in the company statements.

About lorcaserin

• Lorcaserin is an investigational medication that is currently being studied for weight management including weight loss and maintenance of weight loss in patients who are obese or over weight and have at least one weight-related co-morbid condition.
  It is an agonist of serotonin 2C receptor which has been shown to be associated with the control of appetite and metabolism.
  Patents  have issued to cover lorcaserin in the US and other countries.  Most of them will be expired in 2023 without taking into account any patent term extensions or either exclusivity Arena might obtain.
  

MAP Pharmaceuticals announced the US FDA's acceptance of the NDA for Levadex

MAP Pharmaceuticals announced that the US FDA has accepted the NDA filing for Levadex or dihydroergotamine orally inhaled formulation for the acute treatment of migraines in adult.

The US FDA has set the PDUFA date to be on March 26, 2012.

MAP Pharmaceuticals is now eligible to receive a milestone payment from Allergan under the signed agreement.

The agreement was signed earlier this year to allow Allergan take-part in the sales of Levadex.

I'm personally very blessed that I have not had migraine

The FDA issued orphan drug status to Merrimack's MM-398

Merrimack announced that the US FDA has granted the Orphan drug status to its MM-398 which is a nanotherapeutic encapsulation of irinotecan for the treatment of pancreatic cancer.

Merrimack is currently developing MM-398 with PharmaEngine to

Axanum of AstraZeneca received positive opinion for approval in the EU

AstraZeneca announced that the company has received positive opinion for approval from the EMA for its combination product of 81 mg aspirin and 20 mg esomeprazole which it intends to market as Axanum in Europe. 

The company is currently seeking to have Axanum marketed for the prevention of cardiovascular events such as heart attack or stroke in high-risk CV patients who need low-dose aspirin daily but are at risk of gastric ulcers.

If you're not familiar with aspirin and esomeprazole,

Vanda Pharmaceutical signed an agreement with Biotoscana Farma

Vanda Pharmaceuticals announced that it has entered into an exclusive license agreement with Biotoscana Farma SA of Columbia to allow Biotoscana Farma commercialize Fanapt in Argentina. 

Fanapt currently has not been approved in Argentina.  As

Merck submitted the MAA for ridaforolimus

Ariad announced that Merck has submitted a marketing authorization application for ridaforolimus to the European Medicines Agency for the treatment of metastatic sarcomas.  The company anticipates that MAA will be accepted by mid-August.  Once accepted, Ariad is eligible to receive $25M milestone under the signed agreement with Merck.If things go smoothly, Ariad is also entitled to receive another $10M milestone payment once the product is approved and commercialized.

About the agreement:

• Ariad and Merck originally signed an agreement in July 2007 to co-develop

Lilly won in Court against Actavis

Another day, another decision in court, another lawsuit but at least it is not a new lawsuit... rather it's a decision

Lilly (while still in a lawsuit with Amylin) has announced that the US Court of Appeals for the Federal Circuit has overturned the ruling issued by the US District court for the District of New Jersey in regard to the validity of Lilly's method-of-use patent for Stratettera (atomoxetine).

The lawsuit was brought by Lilly

Lilly signed an agreement with Lupin to introduce its products in new markets

Lilly announced that it has entered into a strategic collaboration with Lupin to distribute its insulin products in India and Nepal.  Lupin has agreed to promote and distribute certain products in the stated countries.  The company expects to double the number of sales representatives in diabetes care division.

The following products will be

Diabetes and its market

Sometimes I wonder what is the real battle against diabetes? Is it the food we eat?  Is it the activity we choose to pursue? or is it the drink we take?  In any case, I do feel it's important for diabetes patients to continue to take care of their health and adhere to their medications. the consequence of non-adherence is too great to bear.  Not just for the patients but for the family as well as the healthcare system. 

What is more? Diabetes is no longer the disease of the West.  It's spreading and it's spreading fast.

A couple years ago I visited a country in Southeast Asia and was surprised to

Watson launched generic of Seasonique(R)

True to its word, Watson announced that it has initiated launch of Generic Seasonique (R) which contains 0.15 mg of levonorgestrel and 0.03 mg of ethinyl estradiol.

The launch was initiated after the US Court of Appeals for the Federal Circuit has

Pharmapodia Introduces Orphan Disease / Orphan Drug

Just like a blink, July has gone by so quickly.  Last month has been so busy for me with lots of work and lots of learning.  Although this month will also be as hectic, it might not be as much as the last.  (crossing fingers!)

Due to the work I did this past month, I've decided to integrate my other interest into this blog.

I'm always interested in orphan disease / orphan drug.  So I will try to introduce each of them in this blog.  The number might not be comprehensive but I will try to give as much information as possible.  It might take a couple or a few entries before the information can flow smoothly.  And I'm very excited about this new content development! I hope you do too!

Thank you for your understanding!

I hope you enjoy the new content!

Depomed is to release its financial earnings of its second quarter

Depomed announced that it is to release the results of its second quarter 2011 after the market closes on August 1, 2011.

The company will also host a conference call starting 5:00 PM Eastern Time to discuss the quarter's results.

To access the conference call:

• US/Canada: 888-668-1637
• International: 913-312-1278

Interested parties can join the conference online if phone access is not preferred.

Recent news

Depomed signed an agreement with Ironwood

Depomed announced that it has signed a collaboration, research and license agreement with Ironwood Pharmaceuticals to allow Ironwood utilizing Depomend's Acuform(TM) gastric retentive drug delivery technology for Ironwood's earl stage development program.

Depomed will help with initial product formulation while Ironwood will be responsible for all development and commercialization of the product.

Depomed will receive an upfront license fee and it is entitled to receive additional payments as products developed under the agreement achieve certain clinical and regulatory milestone.  Depomed will also be entitled to receive royalties on product sales.

Financial terms was not disclosed.