Lumacaftor

Synopsis, Chemistry

APIs // Active Pharmaceutical Ingredients

8 API Suppliers, 1 USDMF, 1 EU WC, 2 NDC API, 8 Listed Suppliers

DEVELOPMENTS

4 Drug(s) in Development

4 Drug(s) in Development

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API Reference Price, API Exports, FDF Imports

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3 Suppliers

3 Suppliers

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11 Dossiers, 5 FDA Orange Book, 1 Europe, 5 Canada

RELATED EXCIPIENT COMPANIES

Suppliers, 16 BASF, 10 Roquette, 10 Sigachi Industries, 8 Microlex e.U, 7 Gangwal Chemicals, 6 Gangwal Healthcare, 6 Kerry, 5 Anhui Sunhere Pharmaceutical Excipients Co.,Ltd, 5 Corel Pharma Chem, 3 DFE Pharma, 3 Shanghai Shenmei Pharmaceutical Technology Co., Ltd, 3 Ideal Cures Pvt Ltd, 3 Prachin Chemical, 3 Vasa Pharmachem, 2 Nanjing Well Pharmaceutical, 2 The Dow Chemical Company, 2 Pharmatrans-Sanaq, 1 Ingredion Pharma Solutions, 1 Seppic, 1 Ulanqab Kema New Material, 1 SPI Pharma, 1 Boai NKY Pharmaceuticals Ltd, 1 DKSH, 1 Finar, 1 Aeon Procare, 1 Jai Radhe Sales, 1 Lonza Capsugel

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30 Fillers, Diluents & Binders, 22 Coating Systems & Additives, 19 Disintegrants & Superdisintegrants, 17 Direct Compression, 14 Controlled & Modified Release, 14 Lubricants & Glidants, 14 Granulation, 11 Thickeners and Stabilizers, 9 Solubilizers, 8 Film Formers & Plasticizers, 6 Co-Processed Excipients, 5 Topical, 5 Parenteral, 5 Taste Masking, 2 Rheology Modifiers, 2 Chewable & Orodispersible Aids, 1 Emulsifying Agents, 1 Vegetarian Capsules, 1 API Stability Enhancers

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1 Finished Drug Prices

1 Finished Drug Prices

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2 APIs, 1 DOSSIERS // FDF

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102 US Patents, 8 US Exclusivities

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2 Data Compilations & Company Tracker #PharmaFlow, 63 News

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Novartis leads in new drug approvals; Vertex’s cystic fibrosis med holds highest sales potential

The US Food and Drug Administration’s (FDA) momentum of new drug approvals dropped marginally in 2019 — while 62 novel drugs were approved in 2018, 54 were approved in 2019. FDA’s Center for Drug Evaluation and Research (CDER) approved 48 drugs while another six were approved by the Center for Biologics Evaluation and Research (CBER). Novartis dominated new drug approvals as CDER approved five of its new drugs across five different therapeutic areas. But then, it also made headlines when the FDA raised data accuracy issues over the Swiss drugmaker’s gene therapy — Zolgensma — which sent the company into a crisis management mode. View FDA’s New Drug Approvals in 2019 with estimated sales (Free Excel Available) The gene therapy drug came into the fold of Novartis in April 2018 through the US$ 8.7 billion acquisition of Illinois-based AveXis Inc. Zolgensma — the world’s most expensive drug — was approved as a one-time treatment for spinal muscular atrophy (SMA) in May 2019. However, in March 2019, Novartis learnt of manipulation in the raw data derived from a mice assay used at the time to test Zolgensma samples, but only alerted the FDA in June — about a month after the drug gained US approval. Post that, Novartis came under fire from US lawmakers who said the company should have informed the regulators about the data irregularities before the drug’s approval in May, instead of waiting to conclude an internal investigation. Novartis’ other approvals were brolucizumab (for treatment of macular degeneration), siponimod (to treat multiple sclerosis), alpelisib (breast cancer), crizanlizumab (sickle cell disease) and triclabendazole (fascioliasis, a tropical disease). View FDA’s New Drug Approvals in 2019 with estimated sales (Free Excel Available) Vertex’s Trikafta leads in estimated sales potential At PharmaCompass, we compiled the sales forecasts for the new drugs approved by the FDA in 2019. Vertex’s cystic fibrosis treatment — Trikafta — with expected sales of US$ 3.935 billion by 2024, leads our list. Trikafta is a combination of ivacaftor, tezacaftor and elexacaftor. Vertex already has three cystic fibrosis drugs on the market — Kalydeco (a standalone ivacaftor treatment), Symdeko (a combination of ivacaftor and tezacaftor) and Orkambi (which contains ivacaftor and lumacaftor). Trikafta’s stellar clinical data made the FDA approve the drug within three months of Vertex’s application filing and five months before FDA’s action date. Abbvie’s Humira, the top-selling drug in the world, is beginning to face generic competition in certain parts of the world and two new drug approvals in 2019 — Skyrizi (risankizumab for plaque psoriasis) and Rinvoq (upadacitinib for rheumatoid arthritis) — are critical to AbbVie's plans to grow beyond Humira. The two drugs are expected to contribute over US$ 5.5 billion by 2024 to AbbVie’s top-line. In April, AstraZeneca struck a deal worth US$ 6.9 billion with Japanese drug major Daiichi Sankyo, to jointly develop and commercialize the antibody drug conjugate cancer therapy —trastuzumab deruxtecan. The breast cancer treatment won FDA approval in December and analysts forecast sales of US$ 2.5 billion by 2024 with some believing that the drug could achieve US$ 7 billion in peak sales. View FDA’s New Drug Approvals in 2019 with estimated sales (Free Excel Available) The rise of new drugs from Asia While Daiichi won the FDA nod for its cancer treatment — Turalio (pexidartinib) — new drugs from established Japanese drug makers like Shionogi, Kyowa Kirin and Eisai also bagged the American agency’s approval. Shionogi’s novel antibiotic — Fetroja (cefiderocol) — got approved for complicated urinary tract infections while Kyowa won the nod for its Parkinson’s treatment — Nourianz (istradefylline). Eisai’s insomnia drug — Dayvigo (lemborexant) — was one of the last drugs approved in the year and is expected to generate sales of US$ 329 million. View FDA’s New Drug Approvals in 2019 with estimated sales (Free Excel Available) In 2019, the FDA also approved new drugs from China and Korea. Zanubrutinib (branded as Brukinsa) developed by Chinese biotech BeiGene won accelerated approval for adult patients with mantle cell lymphoma (MCL) — a typically aggressive and rare form of blood cancer. This was followed, almost immediately, by news that the agency approved a new treatment for partial-onset seizures in adults. The drug — cenobamate — will be sold as Xcopri by SK Life Science, the US subsidiary of the massive Korean conglomerate SK Group. Xcopri will provide another therapeutic option to patients with epilepsy. This approval marked the first time a Korean company independently took a compound from the stage of discovery to FDA approval. Xcopri is SK Life Science’s first commercial product and wraps a decade of quiet growth from SK Life Science, which has been developing its presence in the pharmaceutical industry. The company plans to hire a salesforce to support the drug’s launch, which is expected to generate over US$ 1.5 billion in sales. View FDA’s New Drug Approvals in 2019 with estimated sales (Free Excel Available) Sanofi’s controversial dengue vaccine, cast-off drug get approved The FDA very narrowly approved Sanofi’s controversial dengue vaccine — Dengvaxia. The vaccine, which took 20 years to develop, got embroiled in a controversy when in 2017, Sanofi disclosed that Dengvaxia could increase the risk of severe dengue in children who had never been exposed to the virus. In Philippines, 800,000 school-age children had been vaccinated against dengue, the world’s fastest growing infectious disease. The year 2019 had begun with Bristol-Myers Squibb (BMS) and Celgene announcing their US$ 74 billion mega-merger. As the firms worked through the year to get the merger approved, the FDA signed off on Celgene’s myelofibrosis drug Inrebic (fedratinib). Inrebic — the once-daily oral drug — is the first new treatment in nearly a decade approved to treat myelofibrosis. The story of Inrebic’s rise from the dead is a major success for Celgene, which picked up the drug as a part of a US$ 1.1 billion acquisition of Impact Biomedicines in January 2018. Before that, Inrebic had passed through multiple hands. Fedratinib was a flop for Sanofi as patients began to develop a dangerous neurological condition tied to vitamin B deficiency called Wernicke’s encephalopathy. As a result, the FDA put a clinical hold on it in 2013 and Sanofi ultimately shelved the effort. Executives at BMS have said the drug is among a group of potential blockbusters at Celgene. View FDA’s New Drug Approvals in 2019 with estimated sales (Free Excel Available) Our view With over 40 companies from across the world getting new drugs approved in 2019 across 17 therapeutic areas, the horizon for new drug development across the globe continues to widen. Although 2018 was a landmark year for approvals, given all the development activity underway, the day is not far when this record too will get beaten. And 2020 could well witness that day! View FDA’s New Drug Approvals in 2019 with estimated sales (Free Excel Available)

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31 Dec 2019

FDA’s Drug Approvals in 2015: Novel Drugs & New Versions of Existing Drugs

Each year, the US Food and Drug Administration (FDA) approve hundreds of new medications. A small subset of approvals, classified as novel drugs, are considered to be truly innovative products that often help advance clinical care. In 2015, the FDA approved 45 novel drugs, an all-time record high. PharmaCompass has compiled a list of novel drugs approved by the FDA in 2015.The FDA also approved new dosage forms of existing products in the market (email us if you would like a copy), like the 3D printed version of anti-epilepsy drug, Spritam (Levetiracetam). This week, PharmaCompass focuses on the new dosage forms of existing drugs that got approved last year. Modified blockbusters Improving the delivery form of a blockbuster drug is something that not only helps patients but often successfully extends the patent life of the cash-generating drugs for Big Pharma. Here are some blockbuster drugs that saw their modified versions being launched in 2015: Jadenu (deferasirox): With almost a billion dollars in revenues in 2015, Exjade (deferasirox) was approved in 2005 as a tablet for use in a suspension. Novartis, the innovator, got approval in March 2015 for Jadenu, a once-daily oral tablet. Jadenu (deferasirox), a new formulation of Exjade, is the only once-daily oral tablet for iron chelation. Jadenu has simplified daily treatment administration for patients with chronic iron overload. Nexium 24HR (esomeprazole magnesium): Also known as the Purple Pill, Nexium – Astra Zeneca’s blockbuster drug for acid reflux that generated annual sales in America of more than US $ 3 billion – went generic in 2015. In order to extend Nexium’s market, Pfizer and AstraZeneca came together to promote an over-the-counter (OTC) version of Nexium. A capsule version of OTC Nexium was approved in 2014 and is known as Nexium 24HR. Last year, the FDA granted approval to the tablet form of the drug. Iressa (gefitinib): AstraZeneca re-introduced Iressa in the US market in 2015. The FDA had approved Gefitinib in May 2003 for non-small cell lung cancer. Approved as a third-line therapy, in 2010 the FDA requested AstraZeneca to voluntarily withdraw Iressa tablets from the market, as post-marketing studies had failed to verify and confirm clinical benefit. Iressa (gefitinib) is now back in the US as a first-line therapy for a type of lung cancer. However, the patent protection is limited – only one listed patent in the Orange Book which expires next year, and five US Drug Master Files already submitted. Onivyde (irinotecan): Liposomal formulation of anti-cancer drugs have been in vogue for some time. Merrimack Pharmaceuticals got its novel encapsulation of Irinotecan in a liposomal formulation approved for the treatment of patients with metastatic pancreatic cancer, sold under the brand name Onivyde. Vivlodex (meloxicam): In October 2015, the FDA approved 5 mg and 10 mg (administered once daily) doses of Vivlodex™ (meloxicam) capsules, a nonsteroidal anti-inflammatory drug (NSAID) used for the management of osteoarthritis pain. The previously approved doses for meloxicam capsules were 7.5mg and 15mg. Vivlodex uses a proprietary SoluMatrix Fine Particle Technology™, which contains meloxicam as submicron particles that are approximately 10 times smaller than their original size. The reduction in particle size provides an increased surface area, leading to faster dissolution. Kalydeco (ivacaftor): A cystic fibrosis drug from Vertex Pharmaceuticals – Kalydeco – has been making headlines because of its high price (more than US $ 300,000 a year). Price concerns aside, 2015 saw the launch of a pediatric version of the drug as a ‘weight-based oral granule formulation of Kalydeco that can be mixed in soft foods or liquids’. Extended release versions Many of the approvals granted by the FDA last year were to extended release formulations (a pill formulated so that the drug is released slowly) of existing drugs. Kremers Urban’s extended release version of Methylphenidate capsules made headlines last year because of a reclassification of the drug by the FDA. Under the new classification rating, methylphenidate hydrochloride extended-release tablets can be prescribed but may not be automatically substituted for J&J’s reference drug Concerta (methylphenidate hydrochloride). Kremers Urban was almost sold last year. But due to this reclassification, investors aborted their US $ 1.53 billion buyout. Kremers Urban was later acquired by Lannett Company Inc. In addition, extended-release versions of Aspirin, Carbidopa/Levodopa, Paliperidone Palmitate, Tacrolimus and Morphine Sulphate also received green signals for a market launch. First generic opportunities Last year, PharmaCompass shared the names of some drugs which had no generic competition and were also not protected by patents. (Read: “Litigation Free, first generic opportunities list”). Deferiprone (a drug that chelates iron and is used to treat iron overload in thalassemia major) met the criteria. But it still has no generic competitor and is now available as a new dosage form. Amedra Pharmaceuticals, now owned by Impax Laboratories, has enjoyed the rights to sell Albendazole tablets for almost two decades without generic competition in the US. Albendazole is a medication used for the treatment of a variety of parasitic worm infestations. In 2015, patients were provided access to chewable tablets of Albendazole. New combinations at work The FDA also approved multiple combination drugs where the individual active ingredients had been brought to market previously. Most of the combination drugs approved belong to major pharma players like Novartis, Novo Nordisk, Bristol Myers etc. Boehringer’s diabetes treatments – Jardiance (empagliflozin) – approved in 2014 and Tradjenta (linagliptin) approved in 2011, were combined and the combination drug product Glyxambi was approved in 2015. Another combination of empagliflozin, with metformin – Synjardy – was also approved in August last year. Lesser known companies also got combination drugs approved. UK-based development company Vernalis got approval for its cold-cough treatment, Tuzistra XR – an extended release suspension of codeine polistirex and chlorpheniramine polistirex. Similarly, US-based biopharmaceutical startup, Spriaso LLC, also working in the cold and cough therapeutic area, got an extended release tablet containing codeine phosphate and chlorpheniramine maleate approved. Symplmed, a company which is developing various forms of Perindopril, got approval for Prestalia (a combination of perindopril arginine and amlodipine besylate) for the treatment of hypertension. Our view Each year, the FDA approves several pharmaceutical drugs in order to improve patient care; and often versions of these drugs are marketed and distributed across the globe. PharmaCompass’ list of drugs approved in 2015 is now available – just email us for your copy. Accelerate your drug development PharmaCompass has also launched the Drug Development Assistance tool on its platform. Simply search for the drug or the active ingredient of your interest, click on the Drug Development icon on the left menu bar and you can see the inactive ingredients used to formulate the various drug products approved in the United States.

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14 Jan 2016

Find PharmaFlow Article for Lumacaftor

Lumacaftor

Lumacaftor

Synopsis

Chemistry

APIs // Active Pharmaceutical Ingredients

8 API Suppliers

1 USDMF

1 EU WC

2 NDC API

8 Listed Suppliers

DEVELOPMENTS

4 Drug(s) in Development

PRICE INFORMATION

API Reference Price

API Exports

FDF Imports

INTERMEDIATES

3 Suppliers

DOSSIERs // Finished Dosage Formulations

11 Dossiers

5 FDA Orange Book

1 Europe

5 Canada

RELATED EXCIPIENT COMPANIES

Suppliers

16 BASF

10 Roquette

10 Sigachi Industries

8 Microlex e.U

7 Gangwal Chemicals

6 Gangwal Healthcare

6 Kerry

5 Anhui Sunhere Pharmaceutical Excipients Co.,Ltd

5 Corel Pharma Chem

3 DFE Pharma

3 Shanghai Shenmei Pharmaceutical Technology Co., Ltd

3 Ideal Cures Pvt Ltd

3 Prachin Chemical

3 Vasa Pharmachem

2 Nanjing Well Pharmaceutical

2 The Dow Chemical Company

2 Pharmatrans-Sanaq

1 Ingredion Pharma Solutions

1 Seppic

1 Ulanqab Kema New Material

1 SPI Pharma

1 Boai NKY Pharmaceuticals Ltd

1 DKSH

1 Finar

1 Aeon Procare

1 Jai Radhe Sales

1 Lonza Capsugel

EXCIPIENTS BY APPLICATIONS

30 Fillers, Diluents & Binders

22 Coating Systems & Additives

19 Disintegrants & Superdisintegrants

17 Direct Compression

14 Controlled & Modified Release

14 Lubricants & Glidants

14 Granulation

11 Thickeners and Stabilizers

9 Solubilizers

8 Film Formers & Plasticizers

6 Co-Processed Excipients

5 Topical

5 Parenteral

5 Taste Masking

2 Rheology Modifiers

2 Chewable & Orodispersible Aids

1 Emulsifying Agents

1 Vegetarian Capsules

1 API Stability Enhancers

GLOBAL SALES INFORMATION

1 Finished Drug Prices

MARKET PLACE

2 APIs

1 DOSSIERS // FDF

PATENTS

102 US Patents