FDA Accepts Waiver of Clinical Efficacy Studies for Monoclonal Antibody Biosimilars

Professor Sarfaraz K. Niazi secured the first-ever FDA acceptance to waive clinical efficacy studies (CESs) for monoclonal antibody biosimilars. This...

Breakthrough Clinical Results

Professor Sarfaraz K. Niazi secured the first-ever FDA acceptance to waive clinical efficacy studies (CESs) for monoclonal antibody biosimilars. This decision significantly reduces biosimilar development costs (over 90%) and approval timelines (over 70%), enabling smaller companies to enter the market and promoting competitive pricing. The FDA's acceptance aligns with the EMA and MHRA, paving the way for global biosimilar approvals and increased affordability for patients. The first application to benefit from this waiver is a biosimilar to Stelara (ustekinumab).

Key Highlights

First FDA acceptance to waive clinical efficacy studies (CESs) for monoclonal antibody biosimilars.
Expected to reduce biosimilar development costs by over 90% and accelerate approval timelines by more than 70%.
Will empower smaller companies to enter the biosimilar market and spur competitive pricing.
Aligns FDA policy with EMA and MHRA, facilitating global biosimilar approvals.

Drug used in other indications

Ustekinumab Biosimilar Clinical Trials for Non-Psoriatic Indications

Based on a comprehensive review of the available information, there are no specific details about ustekinumab biosimilar agents currently undergoing clinical trials for indications beyond psoriatic conditions. The existing data does not contain information about intervention models for ustekinumab biosimilar trials.

While the original ustekinumab medication itself (not biosimilars) has shown effectiveness in several off-label uses with notable improvement rates:

Alopecia areata (AA): 83.3% improvement in 10/12 patients
Atopic dermatitis (AD): 37.8% improvement in 28/74 patients
Hidradenitis suppurativa (HS): 80.8% improvement in 42/52 patients
Pityriasis rubra pilaris (PRP): 92.6% improvement in 25/27 patients

Additionally, ustekinumab has received approval for Crohn's disease and ulcerative colitis beyond its psoriasis indications. Other conditions where ustekinumab has been used include lichen planus, pyoderma gangrenosum, Bechet's disease, bullous pemphigoid, cutaneous sarcoidosis, cutaneous systemic lupus erythematosus, and vitiligo.

However, it's important to note that these applications refer to the original ustekinumab medication rather than biosimilar versions. The current information does not provide any specific details about:

Biosimilar versions of ustekinumab in development
Clinical trials for ustekinumab biosimilars for any indication
Intervention methodologies being utilized in such trials
Phase distribution of clinical trials for ustekinumab biosimilar agents

While one source mentions ustekinumab in the context of comparing its effectiveness with vedolizumab in anti-TNF-experienced patients with Crohn's disease, this reference does not discuss ustekinumab biosimilars or their clinical trial methodologies.

Company drugs in pipeline

Pharmaceutical Scientist, Inc. Drug Pipeline Indications

Based on a thorough review of available information, there is insufficient data to determine which specific indications Pharmaceutical Scientist, Inc. currently has drugs in pipeline for.

The company's development pipeline appears to be either not publicly disclosed in the provided sources or the company may not have been covered in the available research materials. Without confirmed information about Pharmaceutical Scientist, Inc.'s investigational drugs or therapeutic candidates, it would be inappropriate to speculate about their pipeline assets.

In the pharmaceutical industry, companies typically develop drugs targeting specific disease areas such as:

Oncology (including specific cancers like glioblastoma)
Neurology (including conditions like Alzheimer's disease and schizophrenia)
Immunology (including conditions like systemic sclerosis and inflammatory bowel disease)
Rare diseases (which may qualify for orphan drug designation)

Drug development typically progresses through several clinical trial phases before regulatory approval, and companies often use biomarkers to track efficacy and safety.

However, without specific information about Pharmaceutical Scientist, Inc.'s actual pipeline, we cannot confirm which of these therapeutic areas, if any, the company is actively pursuing.

For accurate and up-to-date information about Pharmaceutical Scientist, Inc.'s drug development pipeline, it would be advisable to consult the company's official website, recent press releases, investor presentations, or regulatory filings which would contain their current portfolio of drug candidates and the indications they are targeting.

MoA used in other indications

Ustekinumab Biosimilar: Indications and Trial Models

Approved Indications

Ustekinumab is currently approved for: - Psoriasis - Psoriatic arthritis - Crohn's disease - Ulcerative colitis

Investigational Indications

Hidradenitis Suppurativa (HS)

Ustekinumab shows promising results for hidradenitis suppurativa, particularly in patients who do not respond to adalimumab: - 70% of patients (7/10) showed improvement in the Physician Global Assessment score - 80% of patients (8/10) demonstrated improvement in the Numerical Pain Rating Scale - A systematic review revealed clinical improvement in disease severity in 76% of patients (34/45) - Symptomatic improvement was reported in 84% of patients (38/45) - A meta-analysis of ten studies (2012-2022) showed a pooled response rate of 67% (95% CI 0.57-0.76) - No severe ustekinumab-related adverse events were recorded

Atopic Dermatitis (AD)

Results for atopic dermatitis were less promising: - A systematic review of 23 patients treated with ustekinumab showed:

Complete AD remission in 34.8% of patients (8/23)
Partial response in 30.4% of patients (7/23)
No response in 34.8% of patients (8/23)
Researchers concluded that the IL-12/23 pathway is likely not an attractive target for AD

Other Dermatological Conditions

Ustekinumab shows promise for: - Alopecia areata (83.3% improvement in 10/12 patients) - Pityriasis rubra pilaris (92.6% improvement in 25/27 patients) - Lichen planus, cutaneous sarcoidosis, cutaneous systemic lupus erythematosus, and vitiligo

Extra-intestinal Manifestations (EIMs) of Inflammatory Bowel Diseases

Effective for arthralgia and psoriatic arthritis in 152 patients
No efficacy found in axial spondyloarthritis
High response rate for dermatological manifestations including psoriasis, pyoderma gangrenosum, and erythema nodosum (65 patients)
Promising results for aphthous stomatitis and uveitis (20 patients)

Intervention Models in Clinical Trials

The following study designs have been employed:

For hidradenitis suppurativa:
Retrospective evaluation of therapeutic outcomes in a multicenter series
Systematic review of published data
For atopic dermatitis:
Systematic review with multivariant model analysis
For Crohn's disease (GALAXI-1 study):
Phase 2, double-blind, placebo-controlled study with randomization 1:1:1:1:1 to different dosing regimens
For ulcerative colitis:
Retrospective, multicentre study across five institutions