Orphan Drug Resources

Orphan Drug Development: Ensuring Best Time to Market

The Benefits of 505(b)(2) for Orphan Drug Development The Orphan Drug Designation Program, created by the Orphan Drug Act of 1983, provides significant financial incentives for the development of drugs for rare diseases. The potential incentives include tax credits for clinical trial costs, waiver of the Prescription Drug User Fee for NDA filing (~$2.4 million […]
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To List or Not to List – That is the Question

A 505(b)(2) may rely on the FDA’s previous findings of safety and efficacy  of an approved drug product. It is possible to rely on more than one approved drug product.  It is also possible that a 505(b)(2) applicant does not have to rely on any approved drug. The correct choice of listed drug product may allow  […]
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Opportunities in Orphan Drug Development for Investors, Pharma and CROs

Orphan drugs, defined in the Orphan Drug Act as drugs developed to treat rare diseases that affect fewer than 200,000 people in the U.S., have begun to make their mark for patients and drug companies. As the number of orphan drugs has increased over the past 30 years, many patients with rare diseases have benefited […]
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Extending Exclusivity: How Long Will It Really Last?

Last week at the Generic Pharmaceutical Association (GPhA) Annual Meeting, the 21st Century Cures Act, a proposed bill with bipartisan support, was a topic of discussion. Specifically, subtitle L—Dormant Therapies, which would offer 15 years of exclusivity for drugs and biologics approved as dormant therapies. From the House Committee on Energy and Commerce’s bill summary/discussion […]
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2014 505(b)(2) NDA Approvals

2014 drug approvals seem to have rebounded somewhat from the past year. In his annual CDER New Drug Review Update, FDA’s John Jenkins cited 35 NME NDA and BLA approvals (calculated through December 3, 2014), up from 27 in 2013 (see chart below). These approvals include 505(b)(1) NDAs as well as BLAs . Some interesting stats […]
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Orphan Drug Exclusivity for a Previously Approved Drug: a 505(b)(2) Conundrum

Until now, if a Sponsor intended to request orphan designation with 7 years of marketing exclusivity for a drug that has already been granted orphan designation, FDA has followed the Code of Federal Regulations (CFR), including the condition described in 21CFR §316.34(c): “If a drug is otherwise the same drug as a previously approved drug […]
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Orphan Designation Granted Letter

Camargo is privileged to work with many clients to develop Orphan drugs.  In order to obtain an Orphan drug approval the sponsor must first request that the drug and indication be granted orphan-drug designation.  This process requires research into both the indication and the potential population.  If the FDA’s Office of Orphan Products Development agrees, […]
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Orphan Designation without Exclusivity: Court asked to decide

Yesterday, Depomed filed suit against the FDA requesting the Court to order FDA to grant their product Gralise (gabapentin) 7 years of exclusivity since it was granted Orphan status; upon approval, Gralise was granted 3 years of exclusivity. Depomed licensed the product to Solvay which became Abbott via acquisition.  Thus the majority of NDA-related regulatory […]
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What is the Pediatric Drug Development Approach for Rare Diseases and Orphan Drugs?

As part of the PDUFA V reauthorization discussions, the FDA and industry are talking about better approaches to rare disease drug development and orphan drugs. Public interests and Congress have mandated that FDA develop new guidances on the required studies needed for NDA approval. In these discussions, participants refer to the “pediatric drug development approach” […]
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Mitosol – an Orphan & 505(b)(2) without clinical studies

Mobius Therapeutics announced that it has received orphan drug status for Mitosol to prevent the recurrence of pterygium after surgical excision. The active ingredient, mitomycin has been used without FDA approval for eye surgeries since the mid 1960’s. Due to its long history of use, the FDA did not require that Mobius conduct clinical trials. […]
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Melphalan – New 505(b)(2) Orphan Designation

Delcath Systems Inc. announced that the FDA has granted an additional orphan designation for melphalan for the treatment of neuroendocrine tumours metastatic to the liver.  Delcath uses a proprietary system they call the Percutaneous Hepatic Perfusion (PHP) System to deliver high doses of existing drugs directly to the site of the tumor.  Melphalan was first […]
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Orphan’s turn 25

I attended a session at the 2008 BIO convention in San Diego celebrating the 25th anniversary of the Orphan Drug Act.  Before the Act, there were almost no orphan drugs.  The exemption from the PDUFA user fees (running $1.175MM today), 50% tax credit for clinical studies and a 7 year marketing exclusivity are powerful incentives […]
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Raptor announces Orphan designation for cysteamine bitartate

Raptor Pharmaceuticals announced that the FDA has granted orphan drug designation for cysteamine bitartrate for the treatment of Huntington’s disease. Cysteamine is currently approved by the FDA and European Medicines Agency to treat nephropathic cystinosis*, also an orphan designation. In addition to the targeted orphan designation, it appears that Raptor is also trying to improve on the […]
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505(b)(2) Orphan Drug Approval with only a BE study?

A reader recently inquired about Orphan drugs and asked me if I thought an Orphan drug developed under 505(b)(2) could be approved based on just a Phase 1 study – a pharmacokinetic or pharmacodynamic comparison to the RLD. At first I thought I couldn’t think of an example.  Generally, an Orphan drug fulfills an unmet […]
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505(b)(2) with Orphan Indication

I was recently asked how you can have a 505(b)(2) application for an Orphan drug.  The reasoning behind the query was that Orphan drugs are new chemical entities (NCE).  This perpetuates the misconception that 505(b)(2) cannot be used for NCEs, which by now readers of this blog know is wrong.  We have discussed the use […]
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505(b)(2) Patent & Marketing Exclusivity

IP attorney Stephen Albainy-Jenai and I just concluded a webinar hosted by DIA entitled 505(b)(2) Patent & Exclusivity.  23 different companies attended, showing the increasing interest in 505(b)(2) issues.  Earlier this year, DIA hosted my overview of the 505(b)(2) drug development process where the attendees had many questions asking specifically about patents and exclusivity, many of which I couldn’t […]
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