By Carla Scaramella – Commercial Assistant

Despite the well-known challenges faced during 2020 due to the COVID-19 public health emergency, the FDA managed to review and approve 53 new drug therapies. That’s not just a number. It´s a considerable amount of new treatment options for patients suffering from a variety of medical conditions throughout the US which also set precedents for all Healthcare Regulatory Agencies across the globe. FDA assessment of safety and efficacy based on clinical trials for these new drug therapies provide the foundation for postmarketing surveillance once these therapies are used in larger -and more diverse- populations. In this way, we can approach the invaluable ‘Real World Evidence’ which is nowadays playing an increasing role in healthcare decisions.

Let’s dive into the approvals and stop on the most remarkable ones, which offer a new and innovative treatment for previously unmet medical needs.

21 out of 53 novel drugs approved in 2020 are first-in-class ones, which means that use a new and unique mechanism of action for treating a medical condition. Therefore, 40% of the newly approved drugs have the potential for a strong positive impact on healthcare.

The following treatments deserve particular attention from this first-in-class-group:

Koselugo (selumetinib) oral capsules is definitely an innovative treatment, as it is the first drug approved to treat neurofibromatosis T1 in children, a rare, progressive and disfiguring disease.

Rukobia (fostemsavir) oral tablets define a new class of antiretroviral medication for those adults living with HIV-1 infection which cannot be treated with other HIV-1 therapies due to resistance, intolerance or safety reasons.*

*To obtain an excel listing with novel drug approvals for 2020 (and their main figures) click here.

Likewise, 31 out of 53 approved novel drugs are used to treat rare diseases -according to US criteria, a disease that affects fewer than 200,000 people-. This means that 58% of these drugs will probably be the first treatment option for patients diagnosed with these rare conditions. Furthermore, as around 70% of rare diseases are genetic, their first symptoms develop during childhood. So we can conclude that 42% of the drugs approved for rare diseases are used to treat children.

The most remarkable approvals to treat patients with rare diseases include:

Evrysdi (risdiplam), the first oral therapy for patients aged 2 months and above with spinal muscular atrophy (SMA), a rare and life-threatening condition that affects muscle strength and movement.

Lampit (nifurtimox) tablets, the first therapy approved by the FDA to treat patients with Chagas Disease -a parasitic disease- from birth to younger than 18 years old. We should underline that Lampit is already registered in several Latin American countries where this disease is endemic, including Argentina, Uruguay and Chile for the treatment of acute and chronic Chagas disease in adults and children.

Orladeyo (berotralstat) capsules, for the treatment of hereditary angioedema.*

*To obtain an excel listing with novel drug approvals for 2020 (and their main figures) click here.

In addition, there were several novel drug approvals across a broad range of diseases, which are likely to improve the below-mentioned conditions substantially:

-Cushing’s disease: the newly approved drug prevents cortisol overproduction with a novel mechanism of action.

INFECTIOUS DISEASES:

-Ebola: 2 new drugs for adults and children.
-Severe malaria in adults and children.
-COVID-19: first medication approved for hospitalized adults and adolescents

CANCER

24 new rugs were approved for the treatment of diverse types of cancer including 2 drugs for prostate cancer (one meant for diagnosis), 11 drugs for lung cancers, 1 drug for tyroid cancer, 2 drugs for HER2 positive breast cancer and a drug for triple-negative breast cancer, the most aggressive form of breast cancer.

In addition, there were remarkable approvals for rare cancers, comprising 2 drugs for Gastrointestinal Stromal tumour (GIST) and drugs for multiple myeloma, neuroblastoma, chronic myelomonocytic leukaemia and other myelodysplastic syndromes, epithelioid sarcoma and metastatic cholangiocarcinoma.

AUTOIMMUNE DISEASES

-Neuromyelitis optica spectrum disorder: 2 new drugs (were approved)
-Multiple Sclerosis’ relapsing forms

CNS and PAIN

-Acute migraine
-Acute pain: an intravenous opioid medication for a hospital setting.
– Parkinson’s disease: treatment for “off” episodes while taking standard-of-care medication
– Alzheimer’s disease: new diagnostic agent to assist physicians in imaging assessment.  

RARE AND DISABLING DISEASES

-Type 1 hyperoxaluria: adults and pediatric patients treatment
-Tyroid eye disease: the first drug approved
-Duchenne muscular dystrophy: treatment for a certain type that begins in early childhood.
-Obesity developed at an early age, associated with pro-opiomelanocortin deficency.
-Hutchinson-Gilford Progeria Syndrome*

*To obtain an excel listing with novel drug approvals for 2020 (and their main figures) click here.

Surprisingly, 16 out of the 42 new approvals that may apply to children are indicated in pediatric population. This is great news, as years ago, most of the pediatric drug use was ‘off-label’, because appropriate pediatric studies had not been conducted so the drugs had not been labelled by the FDA for ‘use in children’. Taking into account that children are a unique population with distinct developmental and physiological differences from adults, this ‘off-label’ use throughout the years sometimes led to inadequate dosing and lack of safety information which placed children at risk for adverse events and denied them potential therapeutic benefits.

Thankfully, over the last years multiple clinical trials on the pediatric population have been conducted and today we can see the rewarding results: several children undergoing critical diseases -whether rare, genetic or disabling- have now a treatment option to improve their quality of life -which is proved to be safe and effective for them-.

In addition to the novel drugs approved in 2020, only a few new dosage forms for already marketed drugs were approved last year. These include, among others, a metoclopramide nasal spray, dolutegravir tablets for oral suspension to treat babies and children with HIV; and also a transdermal system for contraception, containing levonorgestrel and ethinyl estradiol.

This shows that the development of targeted dosing regimens it’s still a challenge for the pharmaceutical industry; even more in the case of transdermal formulation development, for which we can appreciate only one dosage form has been approved during 2020.

Since new dosage forms might improve patient health by increasing patient adherence to therapy, ensure a proper dose is administered and improve quality of life for patients on long-term treatment, it is clear for us in Amarin that transdermal patches are the “stars” that fulfil these attributes in several ways:

• Improving the administration of well-known molecules in a non-invasive, pain-free, self-application manner.
• Avoiding the frequent dosing administration, providing a controlled and sustained drug release for defined time periods -involving a lower peak plasma concentration compared to oral forms-.
• Reducing side effects through avoiding both the gastrointestinal tract -minimizing local irritative effects- and the first-pass metabolism -safer in hepato-compromised patients-.
• Increasing the target patient population, as it can be administered to a patient who is sleeping, unresponsive, or unable to swallow oral medications.

In Amarin, we strongly believe in all the advantages the transdermal delivery systems have to offer for patients and their caregivers. This is why over the past 30 years we specialize in the development and manufacturing of patent-protected innovative formulations of well-known drugs and generic versions of transdermal and topical brand products.

The fact that only one novel transdermal patch has been approved over the last 2 years despite there have been submissions of some novel transdermal systems to the FDA -meaning that NDAs did occur-, confirms the transdermal patch development and manufacturing is a complex and long-term challenge, from formulation to manufacturing, therefore being a niche market on which only experienced players are in.

…And Amarin is proud to be part of them.*

*To obtain an excel listing with novel drug approvals for 2020 (and their main figures) click here.

References:

1. New Drug Therapy Approvals 2020 | FDA
2. Novel Drug Approvals for 2020 | FDA
3. FDA, Paving the Way for Personalised Medicine: FDA’s Role in a New Era of Medical Product Development(Rockville, MD, Oct. 2013)