A picture to understand: the tide of new molecular therapy is coming

A few days ago, a picture from the industry media BioCentury spread in the circle of friends and attracted widespread attention

Approval trends since 2011

Image source: BioCentury official website

Why are new molecular treatments coming out faster?

Why are new molecular treatments coming out faster?

A careful analysis of this picture shows that in many cases, after the first therapy of a new molecular therapy model is approved, there is usually a window period, and then a second breakthrough occurs, breaking the technical bottleneck of the development of new molecular therapy.

After the first therapy of a new molecular therapy model is approved, there is usually a window period, and then after the second breakthrough occurs, breaking the technical bottleneck of the development of new molecular therapy, the speed of subsequent drug approval is significantly accelerated

As early as 2000, the first ADC therapy Mylotarg received accelerated approval from the FDA for the treatment of patients with acute myeloid leukemia

The concept seems simple, but it faces multiple challenges in practice

In recent years, the industry has invested a lot of R&D efforts and resources to optimize the structure of antibody-drug conjugates

The development of bispecific antibodies also entails challenges in terms of therapy stability, delivery mode and manufacturing process

The development of bispecific antibodies also entails challenges with regard to stability of therapy, modes of delivery, and manufacturing processes

The acceleration of the advent of these new molecular treatment models not only benefits from breakthroughs in research and development bottlenecks, but also reflects their advantages in treating diseases

How can new molecular treatment modalities better benefit patients?

How can new molecular treatment modalities better benefit patients?

The advantages of new molecular therapy modalities in the treatment of diseases are reflected in several aspects

In diseases treated by gene therapy, antisense oligonucleotides (ASO) and RNAi therapy, although disease-causing gene mutations have been identified for a long time, they cannot be targeted by traditional small molecule or antibody therapies, and new treatment models provide targets.

Even for targets that can be targeted by traditional treatment modalities, new treatment modalities can provide safer, more effective therapies that benefit more patients

Second, a variety of new molecular therapy modalities have programmable modular design methods, which means that as the technology platform matures, they can be developed rapidly by replacing the modules in the therapy design according to different targets in a relatively short period of time.

Both RNAi therapy and ASO therapy produce therapeutic effects through complementary binding to mRNA sequences

Programmable therapy development is one of the important drivers for improving R&D efficiency in the biopharmaceutical industry

These programmable new treatment modalities also bring hope to patients with rare diseases

These programmable new treatment modalities also bring hope to patients with rare diseases

Cell therapy and gene therapy also offer the possibility of "curing" a disease with a one-time treatment

Looking to the future

Looking to the future

Currently, a number of bispecific antibody therapies are in Phase 3 clinical development, and many bispecific antibodies have entered the regulatory review stage and are expected to be approved in the near future
.
For example, Janssen's bispecific antibody Tecvayli (teclistamab) has been recommended by the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP), and is conditionally marketed for the treatment of relapsed/refractory multiple myeloma
.
Roche's Lunsumio (mosunetuzumab) has been approved in the European Union and is expected to be approved by the U.
S.
FDA by the end of this year
.
AbbVie also plans to submit a marketing application for epcoritamab, a bispecific antibody therapy developed in partnership with Genmab, this year
.

In terms of gene therapy, a number of therapies may be approved in the next year, including Lumevoq (lenadogene nolparvovec) for the treatment of Leber hereditary optic neuropathy, betibeglogene autotemcel for the treatment of beta thalassemia, etranacogene dezaparvovec for the treatment of hemophilia B, Valoctocogene roxaparvovec for the treatment of hemophilia A, et al
.

More new molecular treatment models are also emerging.
For example, CRISPR gene editing is a breakthrough discovery that won the Nobel Prize, and the in vitro gene editing therapy CTX001 is expected to submit a regulatory application this year, becoming the first approved gene editing therapy
.
In vivo editing therapies developed by Intellia Therapeutics have also shown curative potential
.
mRNA technology is expanding into other areas of infectious disease and oncology following its success in the development of a Covid-19 vaccine
.

The in vitro gene editing therapy CTX001 is expected to submit a regulatory application this year to become the first approved gene editing therapy
.

Regulatory agencies around the world have also recognized the potential of new molecular therapeutic modalities and have provided multiple guidance and accelerated development measures for their development
.
For example, the 5-year analysis report on PRIME designation issued by the European Union recently showed that advanced treatment drugs including gene therapy and cell therapy received the highest proportion of PRIME designation
.

Advanced therapeutics, including gene therapy and cell therapy, have the highest percentage of PRIME designations
.

According to the WuXi AppTec database, 23 therapies and vaccines based on new molecular treatment models have been approved by the US FDA in the past three years, accounting for about 150% of all such therapies approved by the FDA before 2019
.
These data show the trend of a surging wave of new molecular treatments
.
We look forward to the advent of more innovative treatments as soon as possible to benefit the majority of patients
.

In the last 3 years, 23 therapies and vaccines based on new molecular treatment modalities have been approved by the US FDA, accounting for approximately 150% of all such therapies approved by the FDA prior to 2019