orphan drug status

ALSO NOTED: FDA OKs Novalar therapy; StemCells files new patent suit; and much more...

Mon, 12 May 2008 06:59:50 -0400

> The FDA has OK'd Novalar's OraVerse (phentolamine mesylate) for the reversal of soft-tissue anesthesia and the associated functional deficits resulting from a local dental anesthetic. Novalar release

> StemCells has filed its second patent suit against Neuralstem claiming two human neural stem cells patents were violated. Report

> Takeda and Affymax have posted more positive mid-stage data for their experimental anemia therapy. Release

> Pipex Pharmaceuticals is reporting that the FDA has provided orphan drug status for oral tetrathiomolybdate as a therapy for Idiopathic Pulmonary Fibrosis. Pipex release

> Forest Laboratories and Daiichi Sankyo have ended a co-promotion deal for Azor, a combo therapy of two antihypertensives. Forest release

> Big-name cholesterol meds are losing ground on two fronts. Report

> Politics are clouding the process of tracing contaminated heparin back to its source or sources--politics that go way beyond the typical Democrats-versus-Republicans grandstanding. Report

> With Big Pharma cutting back, CMOs are picking up the slack. The worldwide market for biopharmaceutical contract manufacturing reached $2.4 billion in 2007, a year-over-year leap of 14 percent. CMO report

> Back in 2005, Swedish pharma monitors raised a red flag on GSK's HIV fighter abacavir and its possible link to heart attacks, The Independent is reporting. Report

> According to India-based Dr. Reddy's CEO, the U.S.'s financing industry is "a complete mess." Report

And Finally... Depressed teens that self-medicate with marijuana only trigger more serious mental ailments. Release

Talecris' antibody drug gets priority review

Fri, 09 May 2008 06:59:54 -0400

The FDA has granted priority review to Talecris' drug Gamunex, meaning the FDA will review its application in six months rather than the standard 10. Gamunex is an IGIV therapy that contains antibodies purified from the donated blood plasma of thousands of people. It's already approved to treat one autoimmune disease, and bring in about $650 million a year. The company is now pursuing an indication for chronic inflammatory demyelinating polyradiculoneuropathy (CIDP), a rare neurological disorder in which patients suffer from weakness and impaired sensory function in the legs and arms. Gamunex previously received fast track and orphan drug status.

- here's the Talecris release
- read this report for more

Related Article:
Talecris backers design a $1 billion IPO

ALSO NOTED: PDL offers bonuses to top execs; Radius earns $28.3M for milestone;Cytokinetics surges on positive data; and much m

Wed, 02 Apr 2008 07:59:50 -0400

> PDL BioPharma says it will pay four top executives nearly $1 million in bonuses through the end of next year to keep them on board as the company undergoes a transformation into an antibody development outfit. Story

> Radius Health has completed patient enrollment into its Phase II clinical trial of BA058 for osteoporosis. That milestone won the company a second-tranche financing of $28.3 million for a total second institutional investment of $67.5 million. Release

> Sirtris has won orphan drug status for its closely watched therapy resveratrol as a treatment for MELAS syndrome. Release

> Pfizer scooped up 87 percent of Encysive stock in a tender offer that closed on Monday. The $2.35 offer values Encysive at $195 million and a subsequent offering will now run through Friday for all remaining shares. Encysive has been developing new therapies for high blood pressure. Report

> Replidyne plans to pay its CFO and corporate development officer $250,000 bonuses to stay on with the company as it completes an unspecified deal. The biotech has been looking to partner up on its antibiotic drug faropenem medoxomil. Report

> Positive data for Cytokinetics' heart failure drug CK-452 helped spur the company's stock price. Mid-stage results demonstrate improved heart function in patients taking two doses of the drug. Final results from the Phase IIa are expected in the second half of the year. Report

> Shares of Kamada jumped 13 percent on the news that its late-stage trial in the U.S. for its Alpha-1 Antitrypsin treatment of congenital emphysema posted positive interim results. Release

> An experimental drug has demonstrated an ability to improve memory and slow the effects of aging in rats. Researchers believe the same drug--S18986--can interact with AMPA in human brains. Report

> Japanese scientists have found a way to deliver a payload of artificial molecules designed to prevent cells from secreting collagen, possibly pointing to a new approach to treating cirrhosis. Report

> The recent revelation in the New York Times that a tobacco company had quietly--some say secretly--funded a research program on lung cancer prompted the Boston Globe to go out in search of tobacco money behind local research groups. It didn't have to go very far. Report

> Intermune announced that an early-stage study of its experimental hepatitis C drug met safety endpoints and appears effective. The news sent its shares up 18 percent. Release

> A federal court struck down new patent rules that had several drugmakers up in arms, including GlaxoSmithKline. Report

And Finally... Should the feds fund studies to compare drugs' effectiveness? The idea appears to be gaining traction. Report

Adherex's melanoma drug earns orphan status

Fri, 22 Feb 2008 06:59:55 -0500

North Carolina-based Adherex Technologies has won orphan drug designation from the FDA for its experimental melanoma therapy ADH-1. The drug is currently in Phase IIb trials in combination with regional melphalan. Adherex is also conducting Phase I trials of ADH-1 in combination with Taxotere and Xeloda.

"Our experience to date combining ADH-1 and melphalan for the treatment of in-transit melanoma has been very encouraging," said Adherex CEO Dr. William Peters in a statement. "To continue with the rapid development of this combination, two additional centers, Lehigh Valley and H. Lee Moffitt, have joined our Phase IIb trial which is ongoing at Duke and the MD Anderson."

- here's the release for more

Related Article:
Adherex takes full control of failed cancer drug. Report

ALSO NOTED: Ablynx pockets upfront fee; Plexxikon publishes cancer data; Solvay drug on fast track; and much more...

Tue, 19 Feb 2008 06:59:50 -0500

> Belgium's Ablynx says it has received an undisclosed milestone payment from Novartis for an exclusive license for a program involved in their alliance on therapeutic nanobodies. Release

> Plexxikon has published data demonstrating that its targeted cancer compound selectively destroys tumor cells which contain the B-RafV600E cancer-causing mutation, a defect present in most melanomas and thyroid tumors and a large number of colorectal and other cancers. Release

> Sanofi-Aventis and UCB have received FDA approval of an oral solution version of the allergy drug Xyzal. Release

> The FDA has accepted Cephalon's NDA for the chemo drug Treanda. Release

> Chelsea Therapeutics says it has reached an accord with the FDA regarding the design of a late-stage trial for Droxidopa, a therapy for neurogenic orthostatic hypotension. Release

> Marshall Edwards has received orphan drug status for NV-196, a treatment for Stage IIB through Stage IV malignant melanoma. Report

> The FDA has put Solvay's Duodopa, the intestinal gel being developed for patients with advanced Parkinson's disease, on its fast track. Report

> Biolex Therapeutics announced that it has entered into an agreement with Merck KGaA to evaluate antibody optimization and production in Biolex' proprietary LEX System. Release

> Behind the scenes at Enhance: Schering-Plough statisticians reviewed some data before the study was complete, according to New Jersey's Star-Ledger. Report

> It was a glitch. That's what the FDA said last week when confronted with the news that it hadn't inspected the Chinese plant that made the main ingredient for Baxter's recalled batches of heparin. Now we know just how big that glitch was: The FDA inspected and approved the wrong plant. Report

> The FDA has finally released proposed rules that would allow pharma to distribute journal articles that support off-label uses of their products--two months after an irate Congressman pulled the veil off the same proposal. Report

And Finally… The Bill & Melinda Gates Foundation has come under a rare attack for its contributions to research efforts targeted at malaria. Report

SPOTLIGHT: Synta, GSK gain orphan drug designation

Mon, 28 Jan 2008 06:59:53 -0500

The FDA has granted its orphan drug designation to elesclomol, an investigational drug that treats metastatic melanoma. The drug is being advanced by a collaboration between Synta and GlaxoSmithKline. Release

ALSO NOTED: Osiris inks $224M contract; Baxter and Nektar to collaborate;and much more...

Mon, 07 Jan 2008 06:59:50 -0500

> Osiris Therapeutics has inked a deal with the U.S. Defense Department to supply a potential $224 million worth of Prochymal for use in treating military personnel exposed to radiation. Release

> Osiris also regained all worldwide rights to Prochymal. Release

> Baxter International and Nektar Therapeutics are teaming up to investigate new therapies for a rare form of hemophilia. Report

> BrainStorm Cell Therapeutics has expanded its collaboration with Rutgers University aimed at curing spinal cord injuries through adult stem cell research. Release

> Jazz Pharmaceuticals has received orphan drug designation for an experimental epilepsy therapy. Report

> Waltham, MA-based Magen BioSciences has acquired the rights to dermatology compounds from Eli Lilly. Report

> Making a living off pharmaceutical companies suddenly isn't so lucrative: IMS Health, the market research company responsible for so much of the hard data on drug sales, is laying off about 10 percent of its work force. Report

> Celgene announced that it expects fiscal 2007 profits to double to $1.05 per share, as sales of its cancer drug Revlimid skyrocket. Report

> As if Amgen didn't have enough to worry about with its anemia drugs under fire. The company is defending itself against charges of bull-whipping sales reps into marketing drugs off label and violating patients' privacy. Report

And Finally... A pharma nightmare with an English accent: The UK government wants to slash prices for prescription drugs by 10 percent. Report

Altus shares dive as Genentech hands back drug rights

Thu, 20 Dec 2007 06:59:58 -0500

Shares of Altus Pharmaceuticals took a dive this morning after the biotech announced that it had reacquired the North American rights to a growth hormone program from Genentech. Altus immediately signaled its interest in a new partner for ALTU-238, with clinical trials set to resume in the middle of next year. Genentech paid Altus $30 million for the licensing rights and now will pay an additional unspecified sum as it transfers the product back.

"We continue to believe ALTU-238 can provide significant patient benefit with better dosing as a potential once-per-week treatment for growth hormone disorders," stated Sheldon Berkle, president and CEO of Altus Pharmaceuticals. "We intend to move this program forward. Altus stock plunged 19 percent on the news.

- check out the release for more information

Related Articles:
Altus loses orphan drug status. Report
Altus inks $140M pact for human growth hormone. Report
Altus stock plunges on clinical trial delay. Report

ALSO NOTED: Pfizer to buy CovX; Florida commits $94M to Max Planck; Affymetrix buys USB; HGS touts animal trial results for ant

Tue, 18 Dec 2007 06:59:50 -0500

> Aggressively looking to expand its role in biologics, Pfizer has struck a deal to buy CovX. No figures were given. Report

> Florida's governor has committed $94 million to bring the Max Planck Society to Palm Beach County, making it a neighbor of Scripps Florida. Report

> Affymetrix is buying USB, which makes molecular biology and biochemical reagent products, for $75 million. Release

> The FDA has approved Forest Laboratories' hypertension drug Bystolic, which it plans to launch next month. Release

> Human Genome Sciences says that animal studies of a new anthrax therapy have demonstrated marked results. Release

> Insmed has received the FDA's Orphan Drug Designation for IPLEX, a treatment of myotonic muscular dystrophy. Release

> Elixir set the terms for its IPO at $14 to $16 a share. Report

> Belgium's Ablynx is touting the results of a Phase I trial of its ALX-0081 nanobody for thrombosis. Release

> Biomarin announced today that it has re-acquired the Canadian rights for tetrahydrobiopterin, including Kuvan, from Merck Serono, The FDA has granted marketing approval for BioMarin Pharmaceutical's Kuvan tablets, a specific drug therapy for the treatment of phenylketonuria. Release

> Medarex will receive an undisclosed milestone payment from Amgen for advancing an antibody into human clinical trials. Release

> Merck and Schering-Plough have responded to public outcry over their plans to re-jig a study of Vytorin, a joint product that combines Merck's Zocor and Schering-Plough's Zetia. Report

> The Internet is buzzing about a Wall Street Journal op-ed piece in which ex-FDA deputy commissioner Scott Gottlieb (photo) championed the sort of off-label promotions that make regulators crazy. Report

> Counterfeit drugmakers have found some unlikely allies: free trade zones. Report

And Finally… A new DNA sequencing technique could dramatically lower the cost to read human genomes. Article

EU, U.S. regulators offer single orphan drug path

Tue, 27 Nov 2007 06:59:57 -0500

Regulators in Europe and the U.S. have joined together to create a common application form for developers seeking orphan drug status. By making it easier to file the regulators are hoping that they will spur more programs for drugs that treat a host of rare diseases. Between the U.S. and Europe there are more than 55 million people who suffer from more than 6,000 rare diseases. But small markets can translate into small profits, which often leave developers cold to any suggestions for new therapies. This rare cross-Atlantic alliance is aimed at making the field more attractive, while giving regulators a chance to gain from their common experiences.

- check out this release from the agencies

Related Articles:
NIH expert sees new drive against rare disease. Report
NIH budgets $71M to study rare diseases. Report
Progeria trial underscores challenge of rare diseases. Report