|  |
Biotechs Scored 23 Product Approvals In 2002, Including 9 New Therapies
The FDA’s been busy the last two months. In December, the agency granted first-time approvals to two important new biologics -- Aralast for hereditary emphysema and Humira for rheumatoid arthritis-- and a vaccine – Pediarix for preventing five diseases in infants. As well, three marketed products -- the anti-cancer drugs Gleevec and Taxotere and the topical psoriasis compound Olux -- garnered the agency’s OK for use in new indications and a fourth – Pegasys for treating hepatitis C virus infection – won the right to be used in combination with the antiviral drug Copegus. Advisory committees voted favorably – at long last – on several of the biotech sector’s most beleaguered drug candidates – Bexxar for non-Hodgkin’s lymphoma and the flu vaccine FluMist.
And the good reviews kept on coming in January: The FDA approved Amevive, the first biological therapy for psoriasis. An advisory committee gave positive reviews on two medications for treating rare diseases -- Fabrazyme for Fabry disease and Aldurazyme for Hurler syndrome.
Moreover, when President Bush turned the spotlight on AIDS at the end of January, the government waived restrictions on a new, 20-minute AIDS test – OraSure Technologies Inc.’s OraQuick – expanding its use from 38,000 certified labs to more than 100,000 doctors’ offices, clinics and counseling centers. Importantly, the FDA accomplished this feat overnight: It received the application for the waiver on January 30 and approved it the next day.
The agency’s also shown unpredicted speed in approving Humira, which was developed by Cambridge Antibody Technology Group plc and Abbott Laboratories. The product was approved in eight months, about two months earlier than expected, and without the need of an advisory committee meeting.
As well, Biogen Inc.’s Amevive garned the FDA’s blessing nearly two months early: The agency had committed to completing its review by mid-March, but granted approval on January 31.
Is this the new, improved version of the FDA for which we’ve all been waiting? It’s too early to say, but the signs are certainly encouraging.
In fact, the agency approved several more drugs and biologics in short order last year. For instance, Hepsera, Gilead Sciences Inc.’s new drug for treating chronic hepatitis virus B infection, sailed through in a mere six months. And Neulasta, Amgen Inc.’s second-generation Neupogen, was approved 10 months after the company submitted its BLA. (For an in-depth analysis of approval times for biotech products, and how they’ve changed over the years, please refer to the Signals article, “Ghost In The Machine.”)
All told, the FDA approved 15 new biotech-related drugs, biologics and vaccines in 2002. Nine of these products were developed by biotech companies, either alone or in partnership with big pharma. The remaining six were developed by pharmaceutical companies.
In the greater scheme of things, biotech companies’ therapeutics constituted slightly more than one-quarter of all new medicines approved by the FDA in 2002, according to the report, “New Drug Approvals In 2002,” just released by the Pharmaceutical Research and Manufacturers of America (PhRMA).
Interestingly, of the nine medicines developed by biotech firms, only four are biologics: Humira, Neulasta, Rebif (Serono S.A.’s beta-interferon treatment for multiple sclerosis) and Zevalin (Idec Pharmaceuticals Corp’s radioimmunotherapy for non-Hodgkin’s lymphoma). The remainder are new chemical entities. (For details of these new products, see the table that follows.)
Biotech And Biotech-Related Drugs, Biologics And Vaccines Approved In 2002*: First-Time Approvals
|
Product Name
(Active Ingredient)
|
Company (s)
(Developer; Marketer)
|
Product Description
|
Indication
|
Approval Date
|
|
Aralast
(alpha-1 proteinase inhibitor)
|
Alpha Therapeutic: Baxter Healthcare
|
Human plasma-derived alpha-1 proteinase inhibitor
|
Augmentation therapy in patients with congenital deficiency of alpha-1-proteinase
inhibitor and clinically evident emphysema
|
12/02
|
|
Avinza
(formerly Morphelan; morphine sulfate)
|
Elan; Ligand Pharmaceuticals (U.S. and Canada)
|
Extended release formulation of morphine
|
Chronic, moderate-to-severe pain in patients who require continuous
opioid therapy (once-daily capsule)
|
3/02
|
|
DAPTACEL
|
Aventis Pasteur
|
Diphtheria and tetanus toxoids and acellular pertussis vaccine adsorbed
(contains 5 pertussis antigens)
|
Childhood immunization against diphtheria, tetanus and pertussis (first
4 consecutive doses)
|
5/02
|
|
Eligard 7.5 mg
(formerly Leuprogel One-Month Depot; leuprolide acetate)
|
Atrix Laboratories; Sanofi-Synthelabo (North America); MediGene (Europe)
|
Sustained release of leuprolide (a leutinizing hormone-releasing hormone
agonist) via Atrigel Depot drug delivery system
|
Advanced prostate cancer
(subcutaneous)
|
1/02
|
|
Forteo
(teriparatide injection)
|
Eli Lilly
|
Recombinant parathyroid hormone; stimulates new bone by increasing
the number and action of osteoblasts
|
Osteoporosis in postmenopausal women and in men, both of whom are at
high risk for a fracture
|
11/02
|
|
Hepsera
(adefovir dipivoxil; 10 mg)
|
Gilead Sciences; GlaxoSmithKline (Asia, Latin America, other territories)
|
Nucleotide analog (blocks HBV DNA polymerase)
|
Chronic hepatitis B virus (HBV) infection in treatment-naïve and treatment-experienced
patients (once-daily; oral)
|
9/02
|
|
Humira
(formerly D2E7; adalimumab)
|
Cambridge Antibody Technology; Abbott Laboratories (Knoll/BASF)
|
Fully human monoclonal antibody to tumor necrosis factor-alpha
|
Moderate-to-severe rheumatoid arthritis in adults who had inadequate
responses to DMARDS (subcutaneous)
|
12/02
|
|
MitoExtra
(Mitozytrex)
|
SuperGen
|
Version of generic drug mitomycin based on company's supergenerics
Extra platform
|
Disseminated adenocarcinoma of the stomach or pancreas (in combination
with other chemotherapies); also for use as a palliative therapy when
other modalities have failed
|
11/02
|
|
Neulasta
(pegfilgrastim)
|
Amgen
|
Long-lasting, PEGylated version of Neupogen (rG-CSF)
|
To decrease incidence of infection in cancer patients receiving chemotherapy
(injected once per chemotherapy cycle)
|
1/02
|
|
Pediarix
|
GlaxoSmithKline
|
Diphtheria and tetanus toxoids and acellular pertussis adsorbed, hepatitis
B (recombinant) and inactivated polio-virus vaccine combined
|
Prevention of diphtheria, tetanus, pertussis, hepatitis B and polio
in infants
|
12/02
|
|
Pegasys
(peginterferon alfa-2a)
|
Shearwater (Nektar Therapeutics [formerly Inhale Therapeutic Systems]);
Hoffmann-La Roche
|
Recombinant interferon alfa-2a modified with PEG
|
Chronic hepatitis C virus infection in interferon alpha-naïve patients
with compensated liver disease; once-weekly injection
|
10/02
|
|
Rebif
(interferon beta-1a)
|
Serono; Pfizer
|
Recombinant interferon beta-1a
|
Relapsing remitting multiple sclerosis
|
3/02
|
|
Remodulin
(formerly Uniprost, a.k.a., UT-15)
(treprostinil sodium)
|
United Therapeutics
|
Analog of natural molecule prostacyclin
|
Pulmonary arterial hypertension (continuous subcutaneous infusion)
|
5/02
|
|
SecreFlo
|
Repligen
|
Synthetic porcine secretin
|
Stimulation of pancreatic secretions to aid in diagnosing pancreatic
exocrine dysfunction and gastrinoma
(injection)
|
4/02
|
|
Zevalin
(ibritumomab tiuxetan)
|
Idec Pharmaceuticals; Schering AG (ex-U.S.)
|
Murine monoclonal antibody that targets CD20 antigen on B cell surface,
conjugated to yttrium-90 (used in conjunction with rituximab)
|
Radioimmunotherapy for treating low grade or follicular, relapsed or
refractory, CD20-positive, B-cell non-Hodgkin's lymphoma and rituximab-refractory
follicular NHL
|
2/02
|
* Includes drugs and biologics developed by biotechnology companies as well as biotech-derived products and vaccines developed by pharmaceutical companies.
Nine first-time approvals for new medicines is not a record-breaker by any means. In 2001, biotech firms won approval for 12 new therapies; in 2000, it was 16. (For a list of FDA approvals in 2001, please refer to the Signals article, “Twelve New Biotech Therapies In 2001.” For a list of the products approved in 2000, see the Signals article, ”New Biotech Medicines: 16 Approved And 19 In The Queue.”)
However, nine more biotech-company products – each already on the market for at least one indication – garnered further approvals in 2002. As well, several other important drugs – including Novartis AG’s rationally designed oral cancer drug Gleevec – were approved for new indications. In fact, both Gleevec and Remicade, Centocor Inc.’s popular Crohn’s disease and rheumatoid arthritis drug, scored two new approvals last year.
§ Aranesp. Amgen’s second-generation EPO has two additional sialic acid-containing carbohydrate chains, which increase the product’s serum half-life. The FDA approved Aranesp in July for treating chemotherapy-induced anemia in patients with non-myeloid malignancies. The agency first approved Aranesp in September 2001, for treating anemia in patients with chronic renal failure, whether or not they are on dialysis.
§ Argatroban. This anticoagulant, a synthetic direct thrombin inhibitor, was approved in April for treating patients undergoing percutaneous coronary intervention who have or are at risk for heparin-induced thrombocytopenia (HIT). Argatroban was developed by Texas Biotechnology Corp. and GlaxoSmithKline plc; it was first approved in June 2000 for the prevention or treatment of thrombosis in patients with HIT.
§ Eligard. Atrix Laboratories Inc. has used its Depot drug delivery system to create several sustained release formulations of leuprolide, a leutinizing hormone-releasing hormone agonist, for treating advanced prostate cancer. The first product – a one-month sustained release formulation -- was approved in January (see the first-time approvals table). The second – a three-month sustained release formulation – was approved in July. Sanofi-Synthelabo will market the drugs in North America; MediGene AG has the European rights.
§ Enbrel. The market for Immunex Corp.'s breakthrough product for treating rheumatoid arthritis (RA) keeps on expanding – in fact, that’s why Amgen plunked down about $10 billion to buy Immunex last year. In January 2002, Enbrel gained its fourth approval – for treating psoriatic arthritis. In June 2000, the FDA approved Enbrel, recombinant soluble p75 tumor necrosis factor receptor linked to human IgG1, as a first-line therapy for patients with moderately to severely active RA. It was first approved in November 1998 for use in combination with methotrexate, or as a monotherapy in patients who have failed other RA drugs. The FDA also approved Enbrel in May 1999 for treating juvenile RA.
§ Gleevec. Novartis’ oral cancer drug Gleevec, a rationally designed signal transduction inhibitor, garnered two additional approvals in 2002. In February, the FDA approved Gleevec for treating inoperable and/or metastatic malignant gastrointestinal stromal tumors. In December, the drug was approved as a first-line therapy for patients with newly diagnosed chronic myeloid leukemia (CML). The FDA initially approved Gleevec in May 2001 for treating patients with advanced stages of CML.
§ Olux. A super high potency corticosteroid formulated as a foam, Connetics Corp.’s Olux was approved in December for the short-term treatment of moderate plaque-type psoriasis in non-scalp areas. Olux was first approved in May 2000 for treating moderate-to-severe scalp dermatoses (including psoriasis).
§ Paclitaxel. NaPro Biotherapeutics Inc. and Abbott Laboratories’ generic version of the cancer-fighting drug Taxol was approved under an abbreviated NDA in May.
§ Pegasys/Copegus. In December, the FDA approved Hoffmann-La Roche Inc.’s Pegasys, a long acting version of recombinant interferon-alpha (which was developed via Nektar Therapeutics’ PEGylation technology) as a combination therapy with Copegus (the antiviral drug ribavirin) for treating chronic hepatitis C virus infection. The agency had approved Pegasys as a monotherapy in October (see the first-time approvals table).
§ Remicade. This monoclonal antibody to tumor necrosis factor-alpha, developed by Centocor (a Johnson & Johnson company), garnered approvals for two new indications in 2002. In February, the FDA approved Remicade’s use (in combination with methotrexate) to improved physical function in patients with RA. In July, the product was approved to reduce and maintain clinical remission in Crohn’s disease patients. The FDA initially approved Remicade in August 1998 for treating Crohn’s disease; it was also approved in November 1999 for reducing the signs and symptoms of RA in patients who have had an inadequate response to methotrexate and in January 2001 as a combination therapy with methotrexate for inhibiting the progression of structural damage in RA patients.
§ SecreFlo. In November, Repligen Corp.’s SecreFlo, a synthetic porcine secretin, was approved to use as an aid in locating and cannulating the minor pancreatic duct in patients undergoing ECRP endoscopy. SecreFlo, which stimulates pancreatic secretions, garned its first approval in April, as an aid in diagnosing pancreatic exocrine dysfunction and gastrinoma (see the first-time approvals table).
§ Taxotere. This product, a semi-synthetic taxol-related compound developed by Aventis Pharma, was approved in December as a first-line treatment (in combination with cisplatin) for non-small cell lung cancer. The FDA initially granted the drug conditional approval in May 1996 for use as a second-line therapy for locally advanced or metastatic breast cancer. It’s also indicated as a second-line treatment for non-small cell lung cancer.
§ Visicol. A new formulation of InKine Pharmaceutical Co. Inc.’s product Visicol (a sodium phosphate tablet) was approved in March for bowel cleansing prior to colonoscopy. It was originally approved in September 2000 for the same use.
Biotech And Biotech-Related Products Under FDA Review 2002*
|
Product Name
(Active Ingredient)
|
Company (s)
(Developer; Marketer)
|
Product Description
|
Indication
(Sought)
|
Submission Type
(Date)
|
Current Review Status
|
|
Aldurazyme
(laronidase)
|
BioMarin Pharmaceutical; Genzyme General
|
Recombinant human alpha-L-iduronidase
|
Enzyme replacement therapy for patients with Hurler syndrome (mucopolysaccharidosis;
MPS I)
|
Rolling BLA
(4/02 - 7/02)
|
Endocrinologic and Metabolic Drugs Advisory Committee voted unanimously
that product is efficacious
(1/03)
|
|
Amevive
(alefacept)
|
Biogen
|
Fusion protein; human LFA-3 IgG1 fusion protein (a.k.a., LFA3TIP)
|
Moderate-to-severe chronic plaque psoriasis (IV or IM)
|
BLA
(8/01)
|
Approved for marketing
(1/03)
|
|
Antihemophilic Factor (Recombinant), Plasma/Albumin Free Method (rAHF-PFM)
|
Baxter Healthcare
|
Recombinant factor VIII
|
Hemophilia A
|
BLA
(6/02)
|
------
|
|
Bexxar
(tositumomab, iodine I 131)
|
Corixa (Coulter Pharmaceutical); GlaxoSmithKline
|
Murine monoclonal antibody against CD20 antigen on B cells, conjugated
to I-131
|
Patients with relapsed or refractory low-grade or transformed low-grade
B-cell non-Hodgkin's lymphoma (NHL); also in rituximab-refractory patients
|
BLA
(7/99);
BLA resubmitted
(9/00)
|
Oncologic Drugs Advisory Committee voted 10-3 in support of efficacy
in rituximab-refractory patients; voted unanimously for use in chemotherapy-relapsed
and refractory, low grade NHL
(12/02)
|
|
Campath
(alemtuzumab)
|
Ilex Oncology; Berlex Laboratories (Schering AG)
|
Humanized monoclonal antibody against CD52 antigen on T and B cells
|
To incorporate long-term efficacy and safety data in label on approved
product
|
sBLA
(5/02)
|
------
|
|
Cialis
(tadalafil)
|
Lilly ICOS LLC (Icos-Lilly JV)
|
PDE5 (phosphodiesterase) inhibitor
|
Erectile dysfunction
|
NDA
(6/01)
|
Approvable letter (4/02)
|
|
Cidecin
(daptomycin for injection)
|
Cubist Pharmaceuticals
|
Antibiotic (acidic lipopeptide)
(in-licensed from Lilly 11/97)
|
Complicated skin and skin structure infections caused by Gram-positive
organisms (including S. aureus)
|
NDA
(12/02)
|
------
|
|
Coviracil
(emtricitabine)
|
Gilead Sciences (Triangle Pharmaceuticals)
|
Nucleoside reverse transcriptase inhibitor
|
Treatment of HIV disease
(once-daily)
|
NDA
(9/02)
|
------
|
|
Dynepo
(gene-activated EPO)
|
Transkaryotic Therapies; Aventis Pharma
|
Human erythropoietin produced via gene activation (inserts DNA sequences
into human cells, thus activating the endogenous human gene to stimulate
EPO production)
|
Anemia in patients with chronic renal failure (both dialysis and non-dialysis
patients)
|
BLA
(8/00)
|
FDA did not accept BLA for filing
(11/00);
Aventis has not yet submitted new BLA
|
|
Eligard 30 mg
(four-month depot)
|
Atrix Laboratories, Sanofi-Synthelabo (North America); MediGene (Europe)
|
Sustained release of leuprolide (a leutinizing hormone-releasing hormone
agonist) via Atrigel Depot drug delivery system
|
Advanced prostate cancer
(subcutaneous)
|
sNDA
(4/02)
|
------
|
|
Enbrel
(etanercept)
|
Amgen (Immunex); Wyeth
|
Dimeric fusion protein; recombinant soluble p75 tumor necrosis factor
receptor (TNFr) linked to Fc portion of human IgG1
|
1) To inhibit the progression of structural damage in patients with
psoriatic arthritis
2) To improve physical function in patients with rheumatoid arthritis
3) Once-weekly dosing
|
1.) sBLA
(10/02)
2) sBLA
(10/02)
3) sBLA
(12/02)
|
------
|
|
Estrasorb
(17-beta-estradiol)
|
Novavax, King Pharmaceuticals
|
Uses micellar nanoparticles to deliver natural hormone 17-beta-estradiol
through the skin
(cream)
|
Topical estrogen replacement therapy for short-term use in reducing
vasomotor symptoms in menopausal women
|
NDA
(6/01);
NDA withdrawn
(4/02);
and resubmitted
(9/02)
|
NDA accepted for review
(11/02)
|
*Includes drugs and biologics developed by biotechnology companies as well as biotech-derived products developed by pharmaceutical companies.
In 2002, biotech companies also won first-time approval for three in vitro diagnostics, one in vivo imaging agent, and a medical device. As well, the recombinant protein used in Wyeth/Medtronic Sofamor Danek’s bone graft was developed by Genetics Institute, now a Wyeth subsidiary. These products include:
§ FortaFlex. Organogenesis Inc.’s bioengineered collagen matrix FortaFlex was approved in March for use in rotator cuff repair.
§ OraQuick. Approved in November, OraQuick is a 20-minute, point-of-care test that detects antibodies to HIV-1 in fingerstick whole blood. Developed by OraSure Technologies, the test will be co-distributed by Abbott Laboratories.
§ Procleix. This combination HIV-1/HCV assay, which was developed by Gen-Probe Inc. and Chiron Corp., uses Gen-Probe’s nucleic acid amplification test to detect HIV-1 and HCV in blood and plasma. It was approved in February for screening donated blood. The test received further approvals in November – for screening voluntary donations of source plasma and for use as a discriminatory HIV test for clinical diagnosis.
§ ViroSeq. Celera Diagnostics’ HIV-1 genotyping system was cleared for marketing in December as an aid in monitoring and treating HIV infection. This in vitro diagnostic sequencing-based test detects mutations in the HIV genome that confer drug resistance.
§ Imagent: The FDA approved Alliance Pharmaceutical Corp.’s ultrasound imaging agent in June. The product, which consists of perflexane lipid microspheres, is used with echocardiography to assist in the diagnosis of cardiac structure and motion.
§ INFUSE Bone Graft: This product combines Wyeth’s recombinant human bone morphogenetic protein with Medtronic Sofamor Danek’s lumbar tapered fusion device. Approved for sale in July, the product is used in spinal fusion surgery to treat certain types of spinal degenerative disc disease.
Although biotech companies didn’t score as many first-time product approvals in 2002 as they had the previous two years, there’s no question that some of the new medications will become best-sellers. These certainly include Hepsera, Humira, Neulasta and Rebif. As well, the markets for the popular drugs Enbrel and Remicade are still expanding, as those two therapies continue to win approval for new indications.
And Enbrel may garner further approvals in 2003: Last year, Amgen submitted three supplemental BLAs on Enbrel – one in psoriatic arthritis, one in rheumatoid arthritis, and one to provide dosing flexibility.
Biotech And Biotech-Related Products Under FDA Review 2002*
|
Product Name
(Active Ingredient)
|
Company (s)
(Developer; Marketer)
|
Product Description
|
Indication
(Sought)
|
Submission Type
(Date)
|
Current Review Status
|
|
Fabrazyme
(agalsidase beta)
|
Genzyme
|
Recombinant human alpha-galactosidase A
|
Enzyme replacement therapy for patients with Fabry disease
|
BLA
(6/00)
|
Endocrinologic and Metabolic Drugs Advisory Committee did not vote
but provided positive review, including that primary endpoint is appropriate
surrogate marker
(1/03)
|
|
FluMist
(Influenza Virus Vaccine Live, Intranasal)
|
MedImmune (Aviron); Wyeth-Lederle Vaccines
|
Attenuated, cold-adapted live virus vaccine (intranasal)
|
To prevent influenza in children and adults
|
BLA
(10/00)
|
Vaccines and Related Biological Products Advisory Committee recommended
approval; for use in ages 5-49
(12/02)
|
|
Fuzeon
(enfuvirtide; formerly T-20)
|
Trimeris; Hoffmann-La Roche
|
HIV fusion inhibitor; blocks virus from entering cells; 36-amino acid
peptide (binds to HIV surface protein gp41)
|
HIV-1 infection (combination therapy with other antiretroviral agents)
(subcutaneous injection, twice daily)
|
NDA
(9/02)
|
Priority review status
(10/02)
|
|
Gliadel Wafer
(polifeprosan 20 with carmustine implant)
|
Guilford Pharmaceuticals
|
Biodegradable polyanhydride wafer containing carmustine (implant)
|
First-line therapy for patients with newly diagnosed glioma
|
sNDA
(4/01);
amendment to sNDA
(11/02)
|
------
|
|
GW433908
(a.k.a. 908 or VX-175)
|
Vertex Pharmaceuticals; GlaxoSmithKline
|
HIV protease inhibitor (prodrug of amprenavir [Agenerase])
|
HIV infection (once-daily and twice-daily dosing)
|
NDA
(12/02)
|
------
|
|
Hemopure
(hemoglobin glutamer -250 [bovine])
|
Biopure
|
Chemically stabilized bovine hemoglobin formulated in balanced salt
solution
|
Treatment of signs and symptoms of acute anemia in adult patients undergoing
orthopedic surgery; to eliminate, delay or reduce the need for red blood
cells in these patients
|
BLA
(7/02)
|
BLA accepted for review
(10/02)
|
|
Inductos
(rhBMP-2/ACS)
|
Wyeth (Genetics Institute)
|
Recombinant human bone morphogenetic protein-2 (rhBMP-2) applied to
an absorbable collagen sponge (ACS)
|
Treatment of open long-bone fractures
|
PMA
(12/00)
|
Medical Devices Advisory Committee recommended approval, with conditions
(11/02)
|
|
Iressa
(gefitinib)
|
AstraZeneca Pharmaceuticals
|
EGFr inhibitor; epidermal growth factor receptor-tyrosine kinase inhibitor
(blocks signaling pathways that play major role in solid-tumor growth)
|
Advanced non-small cell lung cancer in patients who have failed at
least two chemotherapy agents (3rd-line therapy) (once-daily oral tablet)
|
Rolling NDA
(~7/01 - 8/02)
|
Oncology Drugs Advisory Committee voted 11-3 to recommend accelerated
approval (9/02);
FDA delayed approval decision by 3 months
(1/03)
|
|
Istalol
(timolol)
|
ISTA Pharmaceuticals; Senju Pharmaceutical Co.
|
New formulation of timolol (non-selective beta adrenergic receptor
blocking agent, a.k.a. beta blocker)
|
Glaucoma (once-daily topical formulation)
|
NDA
(9/02)
|
NDA accepted for review
(1/03)
|
|
Kineret
(anakinra)
(formerly Antril [Synergen])
|
Amgen
|
Recombinant interleukin-1 receptor antagonist
|
To inhibit the progression of structural damage in adult patients with
moderately to severely active rheumatoid arthritis
|
sBLA
(10/02)
|
------
|
|
LeuTech
|
Palatin Technologies; Mallinckrodt
|
Radiolabeled monoclonal antibody that binds specifically to white blood
cells; for in vivo imaging of infection sites
|
Diagnosis of appendicitis in patients with equivocal signs and symptoms
|
BLA
(11/99)
|
Medical Imaging Drugs Advisory Committee voted to recommend approval
(7/00)
|
|
Memantine
|
Neurobiological Technologies; Laboratories; Merz + Co.
|
Small molecule NMDA receptor antagonist that modulates the flow of
calcium to the brain
|
Moderate-to-severe Alzheimer’s disease
|
NDA
(7/02);
NDA withdrawn
(9/02);
and resubmitted
(12/02)
|
NDA accepted for review
(1/03)
|
*Includes drugs and biologics developed by biotechnology companies as well as biotech-derived products developed by pharmaceutical companies.
These sBLAs are among the 39 product submissions pending at the FDA. All told, there are 13 BLAs, 16 NDAs, one PMA and nine supplemental applications waiting in line (including several biological therapies developed by pharmaceutical companies). Some of these have already been reviewed by the FDA or one of its advisory committees and are awaiting the agency’s final nod. Others have been found lacking for one reason or another, and their sponsors are in the midst of addressing the deficiencies. And a few applications are newly arrived. (For details, refer to the tables in this article. If you would like more information on the review history for many of these applications, please refer to the tables in the Signals article, “Ghost In The Machine.”)
Biotech And Biotech-Related Products Under FDA Review 2002*
|
Product Name
(Active Ingredient)
|
Company (s)
(Developer; Marketer)
|
Product Description
|
Indication
(Sought)
|
Submission Type
(Date)
|
Current Review Status
|
|
Nabi-HB
|
Nabi Biopharmaceuticals
|
Hepatitis B immune globulin (human)
|
To prevent liver transplant patients from becoming re-infected with
HBV
|
BLA
(11/02)
|
Accepted for priority review
(1/03)
|
|
Orathecin
(rubitecan)
|
SuperGen
|
Topoisomerase I inhibitor extracted from bark and leaves of Camptotheca
acuminate tree
|
Pancreatic cancer refractory to available therapies
|
Rolling NDA
(12/02 – 1Q:03E)
|
------
|
|
Photofrin
|
Axcan Pharma
|
Porfimer sodium
(Axcan bought rights from QLT 6/00)
|
Photodynamic therapy: treatment of high grade dysplasia associated
with Barrett’s Esophagus
|
sNDA
(5/02)
|
Approvable letter
(12/02)
|
|
PolyHeme
|
NorthfieldLaboratories
|
Chemically modified hemoglobin derived from outdated donor blood
|
Oxygen-carrying blood substitute for trauma situations
|
BLA
(8/01)
|
FDA issued a refusal-to-file letter (11/01);
company proposed additional trial
(10/02)
|
|
Prestara
(formerly Aslera; GL701)
(prasterone)
|
Genelabs Technologies; Watson Pharmaceuticals
|
Adrenal hormone (dehydroepiandrosterone [DHEA])
|
To limit bone loss in women with mild to moderate systemic lupus erythematosus
who are taking low-dose glucocorticoids
|
Rolling NDA
(5/00-9/00)
|
Approvable letter
(8/02)
|
|
Provigil
(modafinil)
|
Cephalon
|
Synthetic compound that is thought to affect alpha-adrenergic receptors
in the brain
(in-licensed from Group Lafon, which Cephalon acquired 12/01)
|
Excessive sleepiness associated with disorders of sleep and wakefulness
in adults
|
sNDA
(12/02)
|
------
|
|
Ranexa
(ranolazine)
|
CV Therapeutics;
Innovex
|
Partial fatty acid oxidation (pFOX) inhibitor
(in-licensed from Syntex 3/96)
|
Chronic angina
|
NDA
(12/02)
|
------
|
|
Raptiva
(formerly Xanelim; efalizumab)
|
Genentech; Xoma
|
Humanized Mab targeted to T cells (anti CD11a)
|
Moderate to severe plaque psoriasis
|
BLA
(12/02)
|
------
|
|
Replagal
(agalsidase alfa)
|
Transkaryotic Therapies; Sumitomo Pharmaceuticals
|
Recombinant human alpha galactosidase A
|
Enzyme replacement therapy for Fabry disease
|
BLA
(6/00)
|
Endocrinologic and Metabolic Drugs Advisory Committee voted unanimously
that data did not provide substantial evidence of efficacy
(1/03)
|
|
Risperdal Consta
(risperidone; long-acting injection)
|
Alkermes, Janssen Pharmaceutica Products (Johnson & Johnson)
|
Long-acting formulation of Risperdal that uses Alkermes' Medisorb drug-delivery
technology
|
Management of schizophrenia (twice-monthly injection)
|
NDA
(7/01)
|
FDA sent J&J a non-approvable letter (7/02)
|
|
Symlin
(pramlintide acetate)
|
Amylin Pharmaceuticals
|
Synthetic version of human hormone amylin
|
Adjunctive therapy to insulin for treating people with Type I or II
diabetes who use insulin
|
NDA
(12/00)
|
Approvable letter
(10/01)
|
|
Vitrase
|
ISTA Pharmaceuticals
|
Highly purified hyaluronidase
|
Severe vitreous hemorrhage
|
Rolling NDA
(1/4/02 – 1Q03E)
|
Dermatologic and Ophthalmic Drug Advisory Committee meeting scheduled
for 3/17/03
|
|
Xolair
(a.k.a. Anti-IgE, rhuMAb-E25; omalizumab)
|
Genentech, Tanox, Novartis
|
Recombinant humanized anti-IgE monoclonal antibody
|
Moderate to severe allergic asthma
|
BLA
(6/00);
amendment to BLA
(12/02)
|
------
|
|
Zavesca
(OGT 918; miglustat)
|
Oxford GlycoSciences
|
Small molecule inhibitor of glycosyltrans-ferase (inhibits glycolipid
formation)
|
Type I Gaucher disease (oral administration)
|
Rolling NDA
(3/01 - 8/01)
|
Non-approvable letter
(6/02)
|
*Includes drugs and biologics developed by biotechnology companies as well as biotech-derived products developed by pharmaceutical companies.
As mentioned earlier, the FDA’s already gotten off to a great start in 2003 – with the early approval of Amevive, the first biological therapy for psoriasis and positive advisory committee opinions on two medications for rare inherited diseases.
And among the therapeutics now waiting for review and/or approval lie a few potential blockbusters – including the erectile dysfunction drug Cialis developed by Lilly ICOS LLC, the asthma medication Xolair developed by Genentech Inc., Tanox Inc. and Novartis, MedImmune Inc.’s flu vaccine FluMist and Fuzeon, the AIDS drug that blocks viral entry into cells developed by Trimeris Inc. and Hoffmann-La Roche.
It might be a great year – especially if the early signs that the FDA is kicking back into gear turn out to be true.
originally published 02/03/2003 |